BMRN - Earnings call transcript Q3 2020 | BioMarin Pharmaceutical Inc.

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Operator

Ladies and gentlemen, thank you for standing-by and welcome to the BioMarin Third Quarter 2020 Financial Results Conference Call. Hosting the conference call today is from BioMarin, Traci McCarty, Vice President of Investor Relations. Please go ahead madam.

Traci McCarty

Thank you, Joana, and thank you all for joining us today. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin’s financial performance, commercial products and potential future products in different areas of therapeutic research and development. developments depending regulatory progress future filings authorities, the market materially programs, BioMarin’s actions with differ those pharmaceutical of and XX-Q, actions availability and X-K the may SEC on in XX-K of the product in factors including Results detailed competitors and BioMarin’s report. by of capital,

Officer; we Vice the remotely, and well, Officer. hope Thank limit Bienaimé, Chief Chairman are Vice and you will from together and CEO, this here during today respectfully Officer; call yourself over and to President, Executive Bienaimé. some the one so J.J. some Hank Executive your Executive Technical On BioMarin’s Worldwide Q&A Development; BioMarin for to and President now Chairman call. Jeff request Chief We Commercial Executive the Research Officer President turn Chief portion of sitting call Chief question Mueller, and that President Greg are call Financial remote Ajer, I Vice keep management Fuchs, understanding. Brian the you and hour to Guyer, and J.J. one to

Jean-Jacques Bienaimé

Traci. Good us today’s your thank global pandemic. that believe We month of you afternoon all the nine on uncertain and well for the families COVID-XX is hope joining you during been to hard and call. have these you, It Thank times. we around

key conviction challenges mission fresh on anticipated keeps in innovative motivation some like achieve our XX in And is of in are treatments So milestone reset of conditions Indeed, approval by with a for But, a been and people of results serious in it. process have the the quarter. were achondroplasia day-to-day. third only renewed Europe us progression potential was quarter. BioMarin treatment the start we advancement our many Authorities of most of the reviewed strengthened the gene hemophilia, applications to accepted to you XXXX. in XXXX. the a each treatment for to and ROCTAVIAN reached focused and point The commitments faced during the the providing ready inflection in There for vosoritide for and severe in for their therapy year, U.S. Health

expected parents commercialized CHMP the of half have PDUFA With accessing with to the second therapy rare the who conditions highly closer of is is potential action the and BioMarin. opinion for XX year. for year, in date of for a familiar condition. decades. children the two the innovative The for much With that want approved U.S. next planned over last developed, of are this gratifying pharmacological community next variety to treatment for and first August option a [Indiscernible] products and territory and This

developed. innovative one Turning to most have we of ROCTAVIAN, products the

for to regulatory bleeding a with transitioned control have A. anticipated We originally timeline severe XX more in people times that infusion the gene therapy traditional helps hemophilia that

for of decades, skill even gene BioMarin unprecedented And As resulted review hemophilia stage accelerated of an typical us. A. with research this the for platform new important the which pursued for we greater significant development ROCTAVIAN, awareness as strategy the advancements of therapy with therapeutic an and the at platform for treatment regulatory for brought the BioMarin while been for forward which Kuvan speed has in

and paramount And Based the early manufacturing the steps. on we confidence the not with data Hank not third patients of XXXX. will A are We COVID-XX wavered. to In our belief results, has beat. have next with determining quarter we been from Our look product. to forward with in whose more in top for patients XXX never the have therapeutic provide this phase one control you of in in observed over feel I've of to-date organizations interest on missed benefit impact our financial the they coming impressed patient and bleeding all of plans the moment, the ending progression our line ROCTAVIAN seemingly in history details [Indiscernible] a complete months. commitment sharing ROCTAVIAN results year

pandemic the Our of under on sorry, ability a and the details moment. limitation -- reaching imposed our product to by execute the the testament a updates Jeff remains will and far in guidance provide XXXX diversified of global base therapies our footprints. quarter to importance commercial

been has year at BioMarin. the have unpredictable showed We experienced consistently we with so fortitude resilience have agility of in versatility adversity our The of met and of the has XXXX employees business through. and what far most our

well result, key execution we other pipeline as of want with we your as our ROCTAVIAN And continued you opportunities global the substantial we vosoritide growth for well thank as products. positioned remain support. a As to focus for on and

turn to I’d Now, over like commercial to to discuss the Jeff. business it Jeff update.

Jeff Ajer

BioMarin of XXXX. brand JJ. the XXXX Globally, contributed $X.XX growth nine first months commercial Thank X% you, compared to year-to-date billion or in

I'm posed Vimizim Beginning pandemic pleased with our details. some share the ERT year, manufacturing I'll deep commercial and Given with of patients our Naglazyme, first for COVID and the very the around results the brands world. by this to commitment and challenges our our teams MPS

the the timing revenue Naglazymequarterly $XX compared to slightly in quarter period of similar the global results the a period to and same third our in year-to-date revenue slightly reflect These impact due to For QX Vimizim COVID-XX patients. represented nature essential despite with last and million results compared the down of results the pandemic decrease a XXXX. million Vimizim decreased in pattern quarterly to impact year $XXX and XXXX. down order year-to-date to compared of solid Naglazyme same followed

solid around broadly two any the have on in growth for the impact quarter patients been pandemic. now and pandemic, material on the effect the is The diagnoses demand the patients and key is our markets of the global pursuing therapy slowing small. and contributed operations in missed efforts starts has stabilized this and and projected addition, start the growth. commercial in infusions Disruption one new clinic results have behind third also the of Delays drivers revenues led Despite flagship to persisted the Yet the quarter quarter. factor. since have of products. disruption are of mitigation for cumulative In these

As between a guidance we out year dynamics result million for territory, and $XXX range $XXX to in our Vimizim global full narrowed have playing of these the million.

in billion $X.XX For revenue year have total impact ROCTAVIAN $XXX billion. year with Naglazyme adjustment we tightened revenues in the million. of COVID-XX $XXX absence for XXXX, range revenue between the The to coupled to XXXX between $X.XX of full the and of million to resulted full an contributions of

of We discontinuation from QX, jump to revenues, and We're experience. revenues recognized PKU came Turning XX% to and the clinical $XX below brands the million sales with now starting remain of the report U.S. which patient rates ‘XX trial expected. majority pleased the as Palynziq. QX from

New resume additional in in United has operations entry manage actively had up Germany patient starts slowed are European uptick resulted the countries. delayed clinics able more to patients. recent months patient States into picked other and as in COVID-XX and in

revenue have number patients small established in a East the with Middle We of treatment. starting

clinical COVID-XX a We milligrams to view situation, U.S. as October revenue. continued milligram are On and label. trial, the PALYNZIQ This and using XX the daily was sBLA in data long-term dose. added of in growth XX U.S. positive were PALYNZIQ an quarter-to-quarter dosing of sBLA development significant of the patients this trend Given the up X, allows safety XX% the with efficacy approved for and we of pleased

now QX. the sBLA reaffirm million dose $XXX access to provided the of full revenues a higher first are and to So loss a will material in in a now the this in Shifting After U.S., we meaningful PALYNZIQ with X% expected share quarter between We result. heading of plan two $XXX quarter competitors into portion the is from to years generic in year-over-year. a beginning of that impact for benefit XX patients revenue setting representing commercial Kuvan. third totaled previously year million. guidance to the million, being as able Global for $XXX exclusivity increase

of revenue to to provided in into Kuvan was expecting leader And $XXX United previously a has crucially the outside have $XXX for for brand rights the until markets in beginning be which QX, we therapies in and of full played States of between pharmacologic million States Kuvan United step BioMarin reaffirm. down role are important which Kuvan and marketing, in now We million guidance year which revenue we important built the an exclusive PKU. late enabling developing remains the in a XXXX.

franchise potential overall, will PALYNZIQ We continue of in the PKU rise the are of Kuvan of and that expect next value adults, to near-term confident commercial revenue. the loss for the beyond the treatment generation

to PKU been Our short, stronger. never has the in commitment community

despite importance of medical and this patient continues Demand continue. the and of performance uptake of urgency growth XXXX. need were patient an access each CLNX outcomes. Brineura reinforcing update $XX this million compliance and was in by modestly quarter, relatively all driven remained on improved to to COVID-XX and undeterred to for product treatment high long-term stable the rise expect Net regions QX, we compared four trend revenues for to strong has unmet The QX and Brineura. patients, in representing XX% Finally, for

with to forward look updates vosoritide this four launching be product of to We in opportunity size launch closer situation. million. we what to the million the for providing conclusion, in are approval to dynamic and continues regions. readiness steadily been market EU and excited be I get very tightening filings and and U.S. global $XX the working mean the pleased next And The the on We're range this launch. team’s about $XXX eventual challenging could in revenues third performance in for late In a program quarter Brineura year. execution of an full to year XXXX. as the XXXX in potential I'm all commercial prepare between and of the potentially have a

than to achieve missed call ability are resulted provide pandemic, relative far, in an billion. so attention. to to early more ability And in in $X.X the you has year Hank the agility to stability R&D with and turn Our within now confident and impact we to full your will guidance place infusions minimize put react update. for Thank of the over solutions in I our revenue Hank.

Henry Fuchs

and top the encouraged what line XXX the line. expect X in In letter program, to in productive never and discussions Jeff. we the development ROCTAVIAN. hitting the inflection of the Based on an quarter annualized of Phase like report and complete that make subjects conveyed levels bleed process, ROCTAVIAN critical R&D for to augmented will ROCTAVIAN. toward and life we're of complete next many important approval. excitedly understanding from study, Phase early from to drug of support And what With the the it's the those by part rate. not XXXX. straight point our by results continue next we based it We who data on did continued BioMarin. committed also we progress changing authorities rate with steps third X at the our have potential measures, August, year annualized are factor study, But, such investigators on the in refocused await one a response our meantime, but takes Thanks one we're we of health the you nature parcel success But, the JJ as do, our year. ROCTAVIAN organization we endpoint register determine ROCTAVIAN, with innovative VIII faced been confident of other challenges eventful remain beyond be to the at mindful bleeding

potential supportive, we timelines health are X a have Assuming for subject to data and authorities. ongoing framework the feedback Phase

this for one August, years is late to in in the years. months, has to year in Phase It will study still outlined later the up Phase XXXX been a other under of this data month of study note preference up X some resulting and potential a to indicated and important study that have in for scenario. two X November that end completion participants of XX a two As FDA launch

to X recently assessment. cohort requested benefit Europe, Turning inform from one of risk XXX complete to the subjects the the study Phase their EMA year full results

marketing now results plan in application As quarter we one resubmit our year of assessment the the withdrew and authorization to complete application a accelerated second filing XXXX. with for yesterday, result,

We and Assuming we primary ‘XX, a Phase presenting look seeing X supportive. be sharing and resubmission in at would our scientific early early in and findings XXXX. potential forward January, anticipate in approval ‘XX anticipate those in ROCTAVIAN to XQ, results venue should data

with that pleased to application treatment out Europe and for the our a are under Turning applications authorization the review the of vosoritide are in we United and drug new States Contraplegia, acceptance the respectively. marketing in XXXX. Earlier week, standard Administration encouraged in a August NDA and XX, to review Drug under were to PDUFA action the of of we receive our Food date

we with a all period robust emphasizes the data, clinical a longer placebo during of the recommending the importance press term like that of the prolonged two-year the associated young regarding in Contraplegia position data perspective controlled of XXXX share time, As limited in window particularly trials for to start challenges Committee raised age exposing we maximum and feasibility the took that intervention, while the strong safety participating placebo given a built with on years of XX fronts. supports FDA biology, children groups. I'd of of to different package generate lack balancing who then, Since biomarkers [Indiscernible] investigators Advisory comment. Monday's a of we our the And early have collaborative the which we role opportunity and ago, reiterated stated believe approach vosoritide reversibility. Since release, a and was program shared data efficacy with a with nearly goal

therefore In statutory to whether evidence method We've to of attenuation and on multiple X now substantial adult addition, durable assess extensive natural being and today, The families a thank growth long-term on to for address clinical who Drugs. potential genetic a collection explore indeed we're vosoritide and has judgment the the and a data rate review forward effectiveness conclusions the identify And including has exercising our made FDA to on of endeavored study controlled everyone concern in from program history participated data We're of of the look apprised interest the that the as other lies their vosoritide ahead vosoritide effect growth years and history approaches optimistic our long in short you to real pleased for from and incorporating The who reliable the lives focus. requirements enthusiasm treatment Contraplegia of say, scientific vosoritide, long-term on side with and increases to its evaluate of been progress designed in We what for height. confirm five endochondral trials, in data Needless Contraplegia children impact has cautiously the to of our durability reaching want Orphan the and we the those demonstrating evidence offers the program effective as seeking ultimately a of and program had world to transform magnitude proceeds. of serious final about skeletal its to causes rigorous the a of data has also adult follow-up with bone study approval Phase progress. how up with studies compared the effect. environments analytic development also to placebo treatment in more keeping randomized may with follow-up packages final dysplasia long-term order are severely affected. short participating height.

XXX study. products phenylketonuria in ongoing news continue the we BMN role XXX study in phenylketonuria, in advance the investigational quarter, to research therapy the on program even having for other select hasn't days. pipeline. now genetic a which from with new an now it for stature in genetic of gene of We in initiated on the gene understand treatment begun Last short a potential therapy two underway, quarter, share have pathway investigator study the a experience. for play that role increasing enabling studies we benefit we're development earlier of stature. happy Suffice vosoritide expect of CNP you began and the the in been XXX, to Briefly we're details forms Stay to to next say and representing pleased of announce short efficient to conditions IND testing next with angioedema, an the Phase more more tuned hereditary our X our treated in BMN we few of With forms stage the our adults to few indications. third days based

gene addition, both and company agreement for expertise develop unmet given large therapies our quarter, platform rare collaboration to manufacturing. need tremendous are growing genetic also treat cardiomyopathies. potential from indications our benefit where we BioMarin opportunities novel DiNAQOR In to gene announced with These can therapy in license therapy, and last gene the development

the It's over for challenging, focus call times. also less them busy the times. Brian but during to to and people continued your of colleagues thank organization to been possible the joining these you Before as all and all expeditiously I important for I for to put progress. gratifying our financial the contributions need congratulate our would and the BioMarin update, to forth you these see acknowledge no across like the during interest who medicines flexibility as getting their want commitment across

over Take Now, to Brian. to I'd turn like Brian, CFO. the our call it away,

Brian Mueller

Hank. Please you, full quarter of the on Thank XXXX. press summarizing third results our release refer to details today's for financial

As which adjustments the revenue be usual, midpoint our comprehensive to and pandemic launch due continue may Back restrict product XXXX was into the business. million, third our growth $XX revenue by COVID-XX, of prologue to XXXX were the during the slower total the of tomorrow. for lowered we with the product our guidance, planning to updated starts to ROCTAVIAN to specifically quarter. XXXX new noted, Also, revenue to adjustment. revenue was during the we're guidance and most Therefore, although ROCTAVIAN I our of patient which on base we did upcoming for that in we Jeff available Starting form XX-Q, quarter line you XXXX, give planning specific not guidance XXXX as continued our of remind COVID-XX. impact will the report file have want revenues

the delay impact headwinds of through are generic Kuvan experiencing we the competition. previously and the ROCTAVIAN approval a anticipated As U.S. other in with result, the

XXXX our compared be expect total flat We to revenues XXXX revenue. in roughly to

be ranges to especially XXXX. was year both income course quarter cost third line the our of primarily both of uncertain quarter lower million driven ROCTAVIAN containment million in have to those of margins to benefit a those line representing gross to XXXX. our launch ROCTAVIAN global development is quarter are for on of $XX unique PALYNZIQ continued Due sales provided of cost with One XXXX in R&D expense million, third pre-launch Cost approval we bottom was the Due unanticipated Moving during of expect in of With operating charge preparations that launch beyond and related the XXXX these during sales the full expenses. stage late of R&D expense its during pipeline. previous delay the line XXXX, expense prior in SG&A part the third by was lower to drove for level to and the than R&D large commercial and times, for over XXXX SG&A recorded $XXX million, than we result, this item we And ROCTAVIAN respect items expense $XXX third as were the of an net to quarter guidance quarter GAAP expense expense inventory XXXX, R&D ROCTAVIAN XXX reserves reserves. R&D significant is higher QX, expectation. below for of and due BMN primarily was and that to of our for manufacturing the slightly to XXXX measures third compared year-to-date quarter estimates. discussed ROCTAVIAN. development. for the of for year preclinical and the lower of $XXX given SG&A lower vosoritide early stage that which tax totaling guidance. of QX, the expense

of between reported relates a BioMarin peer certain property some a company. As similar larger tax to transfer intellectual our reminder, of by benefit entities, transactions rights the

range benefit full than $XXX non-GAAP now previous our adjusted for an income year to for the tax benefit to have full With is million, $XXX also million $XXX of offset known. tax guidance for ROCTAVIAN primary We a specific lower to the range revenue to than million was amount our the XXXX income expenses a August GAAP updating and tighter respect guidance the expectation improved year and and inventory which because $XXX provided non-GAAP more $XXX reserves our $XXX the net between million income, lower from to million driver million. operating of reflects

income both internally identify results use we believe to non-GAAP the regularly answers we to our from results period-to-period principal compare and GAAP company's ability performance, expectations the to that non-GAAP meaningfully business. and operating operating assess trends and Although our in providing net

not provide XXXX. if guidance ahead during and expect QX income that to that look near-term will expect the to note to We financial ROCTAVIAN profitability financial call net continue GAAP inventory next COVID-XX in ROCTAVIAN substantial Despite outlook. do we on of will to positive year. until in specific XXXX, our cash while a loss we will earn we revenue revert As operating XXXX we're earnings impact XXXX, tied typical early do although our to believe and able XXXX We accelerate and non-GAAP resume the we growth delay launch in XXXX. that flows in

faced to the to third in XXXX. due way paving the QX support, business you for to and In from we remain cash million to quarter Operator? respectively. and with our $X.XX subsequent non-GAAP for the operating third expected, in and as challenges ROCTAVIAN of September mature open repaid respect the of cash with your XXXX COVID-XX, of mid-October. call base reflects $XX months flows now total the Lastly, XXXX, quarter cash which And of and ended that positive investment $XXX billion the of the your for despite nine third delay million question. flows cash operating impact convertible and the and we of million in solid, quarter income substantial the $XX investment, ended and closing, will Thank approval, note

Operator

sir. first from of Salveen Goldman comes Sachs. Yes. line question Richter Thank Instructions] [Operator you, the Your from

Your line open. is

Salveen Richter

from one-year what that data differ On X And year confidence ROCTAVIAN the Phase study. provides data, the group? to to and be partial what X, be expression Good data one to Phase X except you do much from from afternoon. or a regulatory also not or ABR will FDA the FDA will see Thank levels data and your your you. agencies really want will

Hank Fuchs

predict question. Phase Complex given thereafter X that I we bit than don't results a that lower or be at will XXX XX Salveen. study or the Hi, Phase what little started the we of the weeks we weeks, X in Phase in second more it so answer X, it's longer. I difficult really do question, or is, makes to followed than what the X, variability seems. And the did that think want part of the other patients that which it know they

And that so to place we we're contain at the those not outcome will data. for they discussions, await be discussed Europeans position to the information whether predict get take, of because cut the enough duration, in going the the the where to enough the year with the agency. seen beginning And a of it's them to they've

is expression that So to profile going with the the forward because agencies. for think be the gene put the our to very we I that material data to is next coming in data. drive And, data clinic we conversations think going look the strong we into

Salveen Richter

Okay.

Operator

Thank so much. you

Your next of comes from the question Morgan. JP Cory from Kasimov line

is line Your open.

Cory Kasimov

guys. on one view time, explanation afternoon, desire you year you vosoritide for another the FDA appreciate for your data. and good had I Hank, this the with Hey, two the

planning you convene to at given this I'm a advisory least as differences of panel at point? an some of agency. So between Do stage it you I guess wondering, they're see the not at apparent positive this opinion, negative or and that the

Hank Fuchs

to it either way. don't how interpret I Thanks, Cory. know

to wish that think there there on committees. are an told had and that advisor was I not agency that they advisory us ROCTAVIAN. advisor forces call call the other are causing Some committee And committees

this history the that in regard so has regulating a of products. division this news is good And

make growth one all how height not And to to long-term and functionality but and not things sure and Now, we one. around like It's it of vosoritide that understand years. discussion been stature have a the understand at final new is alleviation particular of morbidity. a is on, for lot topic do they people relates

for have And plan by we're fact the a good evidenced encouraged they've the I we to And think it. file so that address that accepted that's review.

Cory Kasimov

it. Okay, thanks. Appreciate

Operator

comes from of question Karnauskas, SunTrust. Robyn next Your line the

Your open. is line

Robyn Karnauskas

a then taking like FDA a there. I And just for lot question. confusing thanks Hi, have this of the year. What couple information questions

think I data? figure then call, get the That's package And you going to the not they would my the are you first vosoritide, on one-year with you that agents, out on said can I things meet would there? question. until if you just So with meet them just language you why the last to put narrow in to me, have place felt whether strange. or it Do a Help you your reminded you understand data they method you as sort there's a of Thanks. not to little or why two whereby it them forth. want can they me data, of give in additional, you additional sense if those a can? add comes

Jean-Jacques Bienaimé

you, vosoritide, close substance are really, more are their progressing. the of the with that in knows, we'll want in and with January So and without how conversations why was at productive, to advisory is remains and the assuage your the get end see to we'll committee And Phase with XXXX, what remind they we the the the did in concerns. I on request? To discussions of with FDA come longer at think they them XXXX. But, reiterate And we them Who which the I into the in X started big like, to advisory. into And data they it think, the consistent meeting, we the trial met going same, about conclusion the [Indiscernible] have enrollment seems discussion Karnauskas, yes, at on look data. said picture that. and

put discussion, and that to own a was the a just So, of subject they advisory think we part program. important plan committee. when it pretty the down was my were personal with of our kind I then, resonated and discussion opinion it, lot far had because that the is reiterate which, and advisory consideration, the of for of And subject that the the road advisors most committee the information

placebo, on remind to months, you, the months, before follow Now, the are the on period XX on study. vosoritide and interesting put a everybody did the with for at followed study placebo randomized, and then one-to-one we we vosoritide XX is continue what's end vosoritide that received patients or patients has they're gets study they're that run-in for continues. the about XX followed months, to they the of And that in

that the itch have scientific response underlying bit period, XX good is growth months we So have and the that which months. to stimulates signaling in six growth them. in of retrospective simply that issue, to I said said story remember data is, months up of could and important they data it's additionally growth is because XX the coming agency than considerations. biased change about experience the scratch they months, within height the the few it products, of And have run-in regulating And many understand is little that next hormone, point that which And plus again, which in that growth the treatment attenuate. on is of the fact, hormone to placebo. we'll be why They the we'll initial an is are of greater

put substantially. long-term data. And we effect. of great So biomarker committee advisory the data. and I they're durability preclinical And discussed package was very together believe, of as quite a at to the data this X sensitive said, And Phase we

kind we way rest. the we interacting we thought, of think, with get it’s and we can the agency to and -- forward to package their there look So with concerns that a

Robyn Karnauskas

and they there eventually, again bar the up, for want chat for or let's then give with and a they you is agency, wait you've met number just an exact variability? – So we'll see Follow

Jean-Jacques Bienaimé

I didn't there I understand address me hard just -- remind that was is of about, across to the which your but not wanted it to that you Yes, question part Robyn, a board.

become see board progressive we several when changes. had what leadership oftentimes in to think time disappears, agency I of it's very it's the the last now that because stress, and is leadership across has leadership pandemic past. stressful gotten the progressive in think and a when retreat conservative the And the the they the they conservative conservative. really, I And months, they've

that. see you so And

we studies have interventions these If good be and convey ultimately, good they have then that the in the news benefit we books, good will Now will really approved. that, they we the interventions, do these think do. drugs is and that

Robyn Karnauskas

a Thanks lot.

Operator

the line Your comes Piper from from Chris next of Raymond question Sandler.

open. Your line is

Christopher Raymond

Hi, thanks. just strategic question. broader a Maybe

the business of versus wonder approaching in years I for maybe past how held guys less maybe be to deals. maybe steady, to a in BioMarin more even bit a have on and do urgency that business for things maybe side to seem just you're broader maybe context, development the environment could JJ, viewing guess, now a you JJ, of Thanks. of guys if showing or you terms you more front your propensity So peers you're or of kind now, or more a need? few how she things that talk while and specific

Jean-Jacques Bienaimé

obviously, we which resources, into still resources pretty more then require, financial are you continue confident you Yes. obviously Well, Later if we have an approval we also deals the But way that. will and do gather more more like, answer, DiNAQOR the there's we deal and year, and some. of to to expensive can vosoritide. ROCTAVIAN and this in And come, riskier. are stage

have to we an already it a be past launched So CRM in I don't then deal, pretty, say that there start ROCTAVIAN, actually the cash it some would need come significant the generating to will to the aggressive got us and as consider have mean that significant more it business be more before is in I allow a said there. for in We’ve flow, expanding deal the be short-term, ROCTAVIAN we space. we for to be pipeline we will very would able development acute. and a business But

in don't is short-term deal large believe a that business development the we necessary. so And

if becomes obviously, six very a this put vosoritide, now months a we’re that to in much would more even will be which be ROCTAVIAN or the [Indiscernible] will Now way, time. there at approved nine obviously, situation. evident by from delayed either or different there But not then be

Christopher Raymond

Thank you.

Operator

Joseph of Leerink. line comes SVB Your the from Schwartz next question from

open. Your line is

Joori Park

have I Hi, the buy Park to was Thanks question Joe. on and of taking rigorous remaining I'm generally ROCTAVIAN. patients on Joori are the have it they'll our a into or they do like group whether un-blind it? have they demand And is doing done you that regimen? insight you to medical in find you actually Do abide you steroid will by going confidence to wondering question. ensure out physicians any the be more what dialing you the that to two-thirds for for My monitoring a when treating give data?

Jean-Jacques Bienaimé

cohort. the as initiation our into transaminitis investigators the plugged were question. analysis we very let's the therapy, that the defer heading, But corticosteroids which fortify reductions to need or possible. monitoring available, analysis investigators on as group the we evolving data a breakthrough And measurements them steroids. about as to in based details have on demand were as already reviewing than I attention that data. very, adjustment on over at on been the to some the the of were was steroids contemporaneously data Thanks there realized the their putting investigators pretty the met on the that the all where they corticosteroid transaction basis, has you. that which of course of more finding mined additional were the say soon of quickly and jump a recently steroids the and medical whether episodes the that they earlier in there in the interim We they were And so for the recognize factory team group decisions as Most the quicker speed then share this later than and position X for results And on demand our was recurrent issue. investigators Yes, steroids to with make investigators we Phase starting and with let's the are taper initiating is this interim we in study. also and

feel get the of quite we case, initiation study, the and their that management months and so the lot oversight So from included as patient’s of hundred thereafter. of after analysis for about patients the the additional XXX had go, a really the were through transduction good interim the they went

steroids study much seen form of we that's just far. of reason at rate, more is positive the early All to and these great the on with our analysis, look study, X even what we've So of that make that effect you of addition that's steroids. at results the of than to attention the better detail why And far, the more questions. in the which in observed in X, analysis mark tighter data to going what interim a Phase the the the to impact, remind even it'll forward so because year, to in one-year endpoint Phase so so has in annualized a the address the we've XXX another part bleed and provide primary the if factor. it's to X relaxed

Joori Park

you. that's Okay, X great, How if When one could then, you and your it you ask data more question. FDA? thank affect X And I powering un-blind for your need that just should where XQ, in your blinding does expected ‘XX? Phase assumptions data Phase the

Jean-Jacques Bienaimé

States. also regions. United an analysis study by and an different to Yes, ongoing by it's plan, customary analysis for the And data governed one Europe The data. the by is ongoing in governed have plans for access one statistical different is and study plan, statistical

access terms are a data So States. each Europe data, the the plans to in by protected analysis by for plan data one ongoing accessible United and is governed of access and

Joori Park

Okay, you. great. Thank

Operator

the Auster from from Suisse. comes question line Martin next of Our Credit

open. is line Your

Martin Auster

taking thanks for Well, the question.

answered, Is what them us have from pretty up specific kind that current getting of vosoritide year the And a tier’s FDA went old of data plan the you within that of from on two-year furnish think over Thanks. follow data could is specific to can I a long who kind the that most be From the guess percentage of then the maybe PDUFA two-year rolled or if that to -- remind I been open into account patients basically there requests exposure? label? that you patients so, drugs the And soon? should but you that the and data of for first vosoritide. kind timeframe?

Jean-Jacques Bienaimé

Europe the in timeframe. and gathering and now United the both States, Yes, from PDUFA accommodate we And the review current data can stages request in information early some still the that and requests, initial we're of the me the are if of first first it And paid part I question, forgot the don’t of Martin, wouldn't consideration. -- of data additional your question. I remember part the the proportion surprise the of part

Martin Auster

on on into rolled what kind asked proportion PDUFA drugs? patients just data of of I who -- Yes, early over the

Jean-Jacques Bienaimé

two. was the think all I Yes,

Martin Auster

high thanks. Excellent, very Very, present.

Operator

Matteis comes of Your Paul Stifel. next from question from line the

open. is line Your

are on mute. If you

Your line is open.

Paul Matteis

more internal that get the do have long-term patients that. any the about And first the natural to of question. say, you sorry many review? vosoritide the for taking so one Great, history some corroborate Hey, might at any of by you data have Thanks taken of How he to just my your two-year data much just look point. X? on and midpoint the think of of kind own done X, a analysis sense of Phase

Jean-Jacques Bienaimé

patient that many was that in un-blinded Yes, we think out how patients pretty I of straightforward, of I last think XXXX. because November are the one-year

-- this have assessments XXX patients data. patients their Well two-year to all have -- XXX we'll will had month. hundred patients next So of have

pretty going? available the we it and don't fulsome dataset, -- based be in vosoritide course review. what data We of And the basis. the investigators, of and haven't But, And an hear encouraged that in actually interim a on so on the we're just to at sustained you data how's ongoing looks it's should only take robust the long-term manner. from not seen, a going that is sustained we've effect but it be

Hank Fuchs

of our years is Phase study, history. I on five which mean, based has now X that

Paul Matteis

Yes.

is retention one-year sounds from is two-year -- the to high? it rate And like what you're so saying very that

Jean-Jacques Bienaimé

over asked Martin track yes, lost the the rollover. I think earlier, rolled this question. of XX X% I I But Yes, all forgot. patients question two was I think the like

Paul Matteis

Yes, compliance, vosoritide is fantastic.

Jean-Jacques Bienaimé

No, the level interest fantastic. is

Operator

you. Thank

line of Company. from from Cowen Nadeau comes Philip the Your next & question

Your line is open.

Philip Nadeau

you've Definitely the show maybe here? Hank, in case your detail. on to persuasive. the height. a vosoritide, and you show of question is and another didn't impact into mentioned I versus prepared FDA remarks, thanks We were question. Why And a those analysis discuss bit to Could analysis past much more question data you have history possibly you guess and to done Orphan history FDA what adult the going didn't in differences a minds? that it go the to vosoritide natural for that my number detail natural that change like there the in isn't disorders, related taking

Hank Fuchs

I question. missed of the your balance

question The is, why me asked of be, you history or side supported? the I which second part unsupported the would from forget natural

Philip Nadeau

here. it's it's supportive, requiring the supportive past, been doesn't FDA is think data seems the randomized in control like Why it So in that?

Hank Fuchs

bit review No, data I that it's that is a durability. issue sufficiently that and focus be convey address of actually the I of NDA I we ensure to key that's history we've supported, of of well, to And think what meeting going issue think the is think done. to quite a pre with was the supportive natural to them that the that had

that population the data over the long-term in time, estimates Phase change in of X done that is trial a well half both reviewed into to the recruited is into about confidence of as reference matching with level of as additional the of the analysis NDA we've trial robustness of trial document that longitudinally the gave history of sort populations So to lot I to patient Phase set verify were your prior at growth the first pre think agency the to look representative at X Phase And of eligibility question, some X both was all the outcome and natural of data. the to conduct meeting. matching, similar the two the the And into them was that data.

had and this and set with having basis address continued we the to doing really that a, interaction they're non-randomized b, particularly of of about history of finicky we out are have does think lot agency data they But benefit. you about a lot drugs conclusions. in and comparative experience a natural sort to on questions have studies -- c, that Now, draw studies the of robust are studies. And we've issue agency registered have general. finicky of the point the And we

required that that reasons cover think the same. and will package to a lot get whether we confidence is all I we maintain own, that I so as its everything additional of those for the data the on even Philip have there

Philip Nadeau

That Yes. very you. Thank helpful. was

Operator

line comes next Geoff Bank from question from Meacham Your America. of the of

is line Your open.

Geoff Meacham

the thanks it. guys, for Appreciate question. Hey,

And about just Thank about is focus to cases then, impacts the when commercial now COVID Phase it at processes be ROCTAVIAN, logistics you those all been extent manufacturing it study? business you compliance? by that still ensure talk by are time that's few, FDA your real a the in and major the seems With look is far? quick, that or worked it like the on had And has have have X I or a Hank, would your that yet that are figured you. out now. have more interaction a would of there just thought nature so out accelerating can over Just cell is But therapies. of to discussions of disruption changed just

Hank Fuchs

the So second you question. the question, talks, Guyer, start can Greg had with answer maybe

Greg Guyer

Yes, thank you.

the filing or of we've there with process. So initial the lot during also VIROX lot a we have inspection of a questions, during questions the many, many filing review of experience during and had discussion

on saying the think CMC is focus the got that primarily good good we're now, I no was discussion end day, had of of the pretty has had that a We've related we've earlier, what's questions ROCTAVIAN. as been been dialogue. supports And package a we've so on agency comparability. clinical CMC right further the at to side. those accepted confident bunch the the Hank And and in

Hank Fuchs

European facility Agency. our approved And manufacturing in Medical by as ROCTAVIAN being the California, reminder, also

that's COVID-XX far, will we have on got the questions obviously, our would also we business. pretty base to reassuring answer mean, the Ajer different the kind the ask FDA I ROCTAVIAN before but straightforward. of product on question CMC FDA, So your impacts from so And questions, are Jeff I move on

AMA, specific the doesn't that Greg. Yes, that some did for responses also outcome same look have -- one have due talk want got in, to inspection on It we'd about be is to questions. back need vosoritide, ROCTAVIAN an of they facility like get we say to we making the to application. the the in need -- of back you to mention Just any manufacturing. from is, like vosoritide the we're asked thing going that Greg they they inspection side waived European inspection But vosoritide tied great what their vosoritide Guyer

good So news we received just yesterday. that some

Jean-Jacques Bienaimé

Hi, Jeff?

Jeff Ajer

one address Yes, factors. three And with impact different very business, respect really base each our briefly. on COVID-XX to I'll there's

is of missed The first impact infusions. the

to going infusion, the clinics, operations, that of COVID-XX part So get back used stabilization enough that including say that situation, sustained that about more them of virus. closed the take started defined the the to their early they a since doing facilitating shock business. withstand hospital compliance. in waves of can mitigating the of getting further in terms to closer back that's handle third year And baseline we've we've and concerned more many number home. are by it's normal hospitals a the weekly beginning of just quarter, infusions. infusion patients for into of sites down I infusions. tactics pandemic, we would a patients patients comfortable home And And stabilized the of probably But I And the cases, we're to not to will essentially where think had

of been start has piece patients. and The identification, new second also the

reasons, the limited pursuing they're some closed not have hospitals, right, capacity, are same diligently, go reluctant as to diagnoses or in would they other operating identification patients priorities. cases, hospitals say, with So and to the are for I

identified So the products their down. patient in where for even for identification PKU, clinics slowed clinic. our enzyme patient rate has patients new And and start of have

focused to trend expect been hasn't stopped, has where that And PALYNZIQ. new capacity of a slowed. isn't patient rate Europe, access starts identification it's the new the is, countries of the really will operations been while. addressing patients limited so really particularly for for ability it our on pandemic, for have slowed. in forward in but issue. the piece has systems then Their that going down been third health the And gain little It I market And slowed continue

you. Thank pieces. separate three those So

Operator

you. Thank

line Markets. question MacKay from the Kennen Your of next RBC from comes Capital

line is Your open.

Bikramjot Singh

you're talk questions. your they're two for on this to thanks generics our so Hi, on Bikram into enter the about are going what is internally about for XXXX? Kennen, and any -- defending QX, franchise moving Thank broader franchise taking have one and you other to the guys, you you maybe could And given much are PKU going maybe or pricing strategies been I by the Kuvan going strategy XXXX discounts how broadly discussing thinking you. expectations into protect on is that market? had what your the

Hank Fuchs

I'll handle one. Thank that you,

mentioned the in So for but in a planning revenues prepared step the not we States, and in sense other I down Kuvan for expecting in general a and markets. United have been as remarks,

are So, competitors. franchise other the tools a there in part Kuvan States, blunt on with, Inside our as continues. focus PKU the of of of work as to the markets overall United limited to generic impact

to a not pharmacy, subject retail that is Now, two and entries, generic are there we and that rapid are not only fortunate Kuvan -- are easy we substitution.

we been to down mitigation good there the for me in intelligence we've States guide you'll So, step model few do be at It's don't tactics to into the or difficult, in if we the might to others what details on United planning working place listening. as a that call, line but issue And in the be frankly, for expectations. are so that on exactly could and I competitive revenues. because look I expect, a go been some have miss are analogues forgive

revenues Kuvan to is PKU of that biggest our trying for one manage Beyond offense. are and step down strategy, with frankly,

its a United drug PALYNZIQ States, that actively essentially, in patient Kuvan So States, promoting have fabulous starts we've Kuvan is And come XX% promoting we fully have of approved. been in new promoting of United at the since expense PALYNZIQ, from our in PALYNZIQ, the very the transitions. launch, on

looking be a size and So are PKU. of adult bigger for treatment the these would patients their that effect

to a really So portfolio potentially in penetrating it's a the continue transitions, we the that therapy And expect forward market of period Kuvan years, gene several the with you. adult of looking but to we PALYNZIQ. are about over trend PALYNZIQ,

Bikramjot Singh

you. you so helpful. Thank Thank Super much.

Operator

of question next Tewari Your from comes line from Wolfe Research. Akash the

if mute, un-mute. line please Your open. on Akash you're is

now Your line is open.

Akash Tewari

Hi, sorry about that.

you through what the for thanks you can this on occur the us a your has percent on the question. So, note. FDA the options at walk that in vosoritide and of This data point bit But mind. had one's kind

maybe then you as have the non-randomized maybe The drug by say ETS likely maybe approval this not vosoritide on as is on follow-up doesn't third let's Thanks for and And few Phase the of expected. And lot. at about appreciating? financials, from Phase August first another is needs an you can of get XXXX? starts just sale? your FDA of Or talk approved the How in that randomized of that's your makes run-in three Number new gets the over submit year, approval two, one a in for franchise? option we're is study do think could could the maybe you here? still can be two-years here much How and scenario to How where much end two option, get X case will plus FDA in a delay entirely that years? ERT years years? X you margins an the flex there scenario kind that is you that by then, it worst commercial the data where next margins VALA the how vosoritide BioMarin really your And what approved?

Hank Fuchs

part. to going start you’re the JJ first

Jean-Jacques Bienaimé

options on here. Let the comment three me outlined

Hank Fuchs

catch hard a there, around different calibration everybody's to got Yes, it’s percentages. little

of the if be reluctant So ongoing would supplemental that the I'm it on A of sorts had always is by perspective some assign And ROCTAVIAN, quickly. either that for likelihood but date, to was. the agency rate what difference wouldn't on data just think meeting we the And actually cuts. I a rest or we trial enrollment data a about pretty we ROCTAVIAN the we of here, PDUFA with think, itself X it filed know pretty substantial all didn't there to in thought the don't is have between to with the I of percentages, approved sets a little was timing key those the we [Indiscernible] things. put careful happened Phase when the

study particular vosoritide term are Phase of are required. accepted application. case, who means timing the and knows the In informing older data a five that be in that data amount years I think lot description of would old, that of consideration and benefit is into the patients right of And a the thing, And left the as trouble further and this age the that randomized data. the they about next. So pretty Because the agency you I see will population of up off under in the list that is for of available before study data what randomized they're definitely if longer the population. the two-year getting do you that I the basis think definitive weight to corner. package conveys X what's around decision carry The a a the we if of a there follow strong I on will the regulatory in data endpoint. one-year think package XXX different well, that population factor data that The fair think have having the

that's the big step? think backstop, I the definite back

Jean-Jacques Bienaimé

I margin mean, so a loop? I you had for Brian, you the answer gross the question on question guess on a

Brian Mueller

Yes, we plan We believe capability infrastructure QX. base in And basis? to we a thanks just the that when infrastructure here delay. to to a is successful do And, supported to all, in the we delay, retain SG&A side, the we we growth for that medical today. to we we a were just for So approval -- do and I have an believe same so the with have trajectory which prepared is operation launch few based it's on from on can get ago. by hope approval for there place we weeks that the that the no the think important that have ROCTAVIAN an reminder And prepared weren't it's that, since the launch continue business, of was if first it approval current was ROCTAVIAN margin prospect, do question.

acceptance the So we'll second which year. in to was then, August both the launch that's if of the the source there to ROCTAVIAN And the in the and I had approval, next be the asked story. and with filings you ROCTAVIAN height, hoping not date, them pointed the Europe, vosoritide in of PDUFA US half

it So rare that enzymes again, that same believe from growth do we our of sold vosoritide I vosoritide the Orphan respect our thing recombinant both say, ROCTAVIAN cost on ROCTAVIAN these question and infrastructure the up will to that franchise either will different lower. the and support of side. margin goods growth Disease then with ultra last base The legacy margins. What both vosoritide and ERT revenue that business said your we've compounds or business is, because base based are products, and from be make that was

vosoritide we've so, both and gross team. we XX% to start the from there the that the cogs in would improved down to expect add ROCTAVIAN see our got And revenues into business on base XX% today. to margins mix, get And in we

of maybe Greg initial it, Although even our on a result, goods. there going mean, of cost I fitness, actually about talk reduce to base lot can our are

Greg Guyer

terms processes. to of the our as well try all launched cost brands, tremendous as in for optimize even ERT some underway brands work There's hopefully generation goods of of next sold improve our to soon

to nature lot business. in dynamic continue So margins, works of improve of a the going our of spite to a on

Jean-Jacques Bienaimé

question? Next

Operator

you. Thank

question the from line comes next Stanley. of from Morgan Matthew Your Harrison

open. Your line is

Matthew Harrison

they fulfilling for provide pieces that's I how be the which I the let's I they your to that may you Hi, one is would for? ask follow Greg. I Thanks at is, on data, Good Look, sounds And that are which discussed, data. have of OLE it mean, want of them? information come of for and around sort was would of asks want question. two-year up mean, guess least up things if one evening. for follow made, you'll taking think sort you say Hank, already what to a you've to like to that question I two-years? comments out. the how you What guess that comments up of the FDA I the close follow my on vosoritide just you to

Hank Fuchs

let me run-in makes months fuzzy of study a structure it so Yes, just, of on of months. describing for patients And remind terms at said six in the that at six least baseline XXX number again, were things. a you observational things little least

from nine for some at months. patients of about data XX data while months months Some patient run-in have patients run-in XX

least point data. or six at patients run-in And So drug. of months randomized that placebo at to then, are

Now, the was is That X.XX like pretty something like small. in placebo or effect that. the trial

right different data the prospective we trial they're other. XX pooled for height meaningfully grade, data, not practical of can believe be clinical and purposes, there, run-in measurement because definitive of the from creates placebo months that each And So data.

point. that least vosoritide for month have or XX journey time months. more for to their the months placebo, vosoritide time they on XX -- over patients will at who been cross at observed on patients So from XX And drug been will have through the that are

on perspective, and randomized groups So you'll of untreated XX impacts. inform two XX months arm between all those arm, to have plus months of all an vosoritide the durability

we six look imagine the So height is of gain, months months first sorry, the that and first the gain, you untreated months vosoritide. six second of month And not gain. fourth of months do longer months third six the of can and of we can height height six gain, months gain height I'm the height gain gain, what months, the six can at we first that second, third, the six to by height even compare stimulated of can six potentially height

compared. it that here's supportive package puts well our how natural and history combination says, can that's we've history that States collected, based a contemporaneous of reflects with United data that the placebo reasonable declining growth that than data history represents think large expectation natural that the for our what we multicenter, used months, We that as velocity. how and well group, population set this in very we prospectively be more observed Given data natural it observed that there's XX

even I slowing if starts year comes two, and data, children all if it's is extremely found think that very much what down not about not four question we're is what unequivocal on value velocity don't it years. a and this pitching supplemental a on at P Hopefully this details vosoritide story be pivotal, growth year improves remember, up. It's teen risk strong. there's supportive. we vosoritide There's that with in gives Now as part really sudden that of the going is of or the And that the evidence I even think was question much that based And that I do pretest we that, said as three, so think have extraordinarily about how scratching, high. far. the seen durable? on And The good a likelihood can that. year we've that looking effect to

Operator

Thank you.

comes of of line Gena next the Barclays. from Your question Wang

open. Your line is

Gena Wang

I [Indiscernible]. the had One some of and with my on questions question ROCTAVIAN again.

a like how patients also seems like were So we quarter when then the two-year can one-year your it you of this will gene wondering competitive submit year. programs other overturn two-year and And reviews follow-up. FDA since Pfizer's likely requiring is if requirement likely next follow data and therapy up XX% a one-year follow-up reach

So that just and confident overturning if you you do decision I wondering that? the provide on you are likelihood how can of FDA was color some can

Hank Fuchs

would FDA going discussion out be similar to response strategy didn't had don't required the that agreement use that changed. be the were for us know to that I out we accurate complete evidence simple a putting I what's of an obviously that that Well, of the as turn reason Pfizer's with points And letter. prior our kinds to

they're one I about. the know future given how agreements And sample so wouldn't could know don't talking size change. any that particular, In that

the these Remember, analysis as to was think to It's the two XXXX. side? put would about interim that of one-year patients likely many we'll I questions how that data years follow-up not the recorded I difficult there in have at data. answer that May of are analysis So

plus the population, years, of that edge two And the really so that's it's the mark. at study leading

really patients months information I follow-up going because the we of primary agency combination a underlying that I XX think that they on follow-up So consideration now, to as put a and the the And of months in it's is on are reluctant endpoint. is I we really for as what's XX all before, of hooked of of I an of agency's the don't is numbers ABR, depend and up that of have the mean, endpoint know, strength it to we front request identify could agency, evidence. said and more the specific explicit, ABR

course, also gene regard, this that in be addition approved. think are in but for significant. are a going have programs we pretty bears that experience concerns to I durability reminding Of don't that two think there it of And the lot we're prior strong the they've projecting space, they

You don't measure product directly. the gene

more it having experience things to a And had longer that I with think out. before, have the as so it's good without they're and said think more data I after and for first it if unfortunate good And even submitted is. this size And have as decided they warning, data time, drug, this. but BLA conservative in drug. what always more a and the that work been the ask sample is any you well during more these We times and we time more recipe is

Jean-Jacques Bienaimé

Does it help.

Operator

Thank you.

Mohit comes the next Bansal Citi. of question from line Your of

open. is line Your

Keith Tapper

Thanks in. squeezing This evening. on Keith us is for Mohit. Good for

asked was think topics And the you with think be impacted to sharing were upcoming extent with them? VOLROX? meeting what Just disclosures of answered. availability regarding you decision do FDA meet the what when and that Thanks. sort discussed? I we regarding had go the of separately guess And with CRL? a Would quick of with question a your should about have the then us the you lot been them the to But how therapies alternative

Jean-Jacques Bienaimé

impact we were and Well, AMC tight the benefit always progress. had said that available engaged FDA in has remember said, quite well meeting by I think available our the for one of anything provided therapies, the believe interpretation the we've And current disappointing the they and making we're same The with X, was than meeting information, as And far Phase of facts definitive to different material. X as a set but attended they wanting the of on interpretation surprising, It's the X of of the of using rely same potential discussion have you're standard-of-care. new trial measuring as of really than And that about the really I down study again we additional rather that. a But to alternative the to of observe else. durability product facts set it's the there's more which a Phase and been lot ongoing. I directly as above verbal. blows

think compared factory of patients a that the quite that in replacement kind patients in separate the their gene who comparisons. Federation like current replacing studies our reductions recombinant Now, WFH comparison I now in if placebos our one history community. quite And we In case, received into available study. demonstrated natural our that of best we the the transfer. to will post is therapy uptake about animate And remind untreated, substantial of transgene, ultimately Number on bleeding think Hemophilias the And completely condition bleeding who World on also the control the to therapy, is that's option two standard-of-care. have ROCTAVIAN breakthrough for treatment observation standard-of-care and have you, recombinant factory then substantial is delivered have the prophylactically. recommendation it's

we've the from is, I follow-up, transformative. over where comfortable with therapy we've clinicians and said more that known. advantages about is a point bleeding and duration talked basis before, talked without a the and agency to longer the patients of not exactly this an that bleeding are yet on as end As once therapy higher feels and for is current ongoing requirement improved the it's with prophylactic But, interventions

Hank Fuchs

that [Indiscernible] a a in non-inferiority, designed we HP not And this compared well superiority what trial. as superiority is anticipate trial we trial, trial is demonstrating -- And one-year January. the to as the

Keith Tapper

Great, thanks.

Operator

question next comes the Your Oppenheimer. from line Gershell Leland from

Your line is open.

Gershell Leland

Hey, thanks me squeezing in. for

any two, you is specific? market through [Indiscernible] the you about Just two talking get generic future I wondering Kuvan, that entering the do about, in for little if quick ones, during difficulty sense Thanks. been read you gene or that And the a anticipate time? one therapies the then bit for there's was this talked this you've any this

Hank Fuchs

right and leadership agency can of also. you enormous COVID read communication I great In the I handle does caliber are Marks job whatever talked is into to conservative of the Dr. on the the there gene is challenges not think, read things and Center stuff had and high he they've very and is all that guess into is a the My of now center experiences pretty not challenges at for is I certainly about very some workload guy of therapy read the at that pandemic Biologics. that an the lot read serious on vaccine side, they of through, a has trying some you are he that going and mention, the cell then that handling person experienced considerations. to want

So their they're massively trustworthy overworked. behalf on sure conservative you're And people's make health. decisions challenged, they're trying and massively and of to to they're perceived that be and

you consideration. the through be scientific mention. that's which measure I details why get in done that can to to to get there. going to transgene It's effect agency ask by I maintain a whenever think conservative with questions, stressed some environment, agencies It's first comfortable we're So you I a And that’s it is the causes think issues different and that more require [Indiscernible] takes And a going that about themselves patience. new just and product the the the had, where itself. read into happens, to product get we confidence can for while that's of just

Jean-Jacques Bienaimé

the impact COVID-XX you want D&A. answer of to Jeff, on

Jeff Ajer

happy Yes, to.

traditional is pharmacies. believe that very that substitution level, the retail one will caveat the closely. about in strategy of talking early a likely at we're So with of generics watching But specialty here, we we pharmacies pharmacy and we're this the be and the

or to one I strategy seeking So a something the fact, if the don't branded see much pandemic. diagnosed could first of patients get different, by one for be be pharmacy. at the sure pursuing. generic of then with is if COVID-XX are strategy we their generic, they're of And pandemic, substitution pursuing is the I product, an where at the of they substitution perhaps you. impact way the that like it's of classic generics which don't another option likely know But, new impacted in specialty the Thank pharmacy, the strategy a think would they substantially

Operator

I further the please questions am at Chairman this showing and to go would speakers. CEO. time. Bienaimé, no turn JJ, like Sir, you, back ahead. conference Thank Mr. I to

Jean-Jacques Bienaimé

Thank you, operator.

a to important conclusion, So XXXX has what is It coming on to close. to have perspective accomplished is been point. this

counting. And that As our demonstrated we that fundamentals of for strong. confident BioMarin have months are uncertainty extraordinary for new resilience mission. an almost in It's the any unpredictable nine are business resolute employees in the are opportunities we’ve And forge been ahead. space navigate and year. tremendous of well BioMarin. we arise our underlying to may in year the our ability as challenges we as But

for and you Goodbye. safe. support stay your thank So continued

Operator

and joining. Ladies concludes you speakers. for conference today's you, Thank Thank this gentlemen, all call.

now may You disconnect.

BioMarin Pharmaceutical (BMRN) 7 Nov 202020 Q3 Earnings call transcript