Earnings call transcript Q2 2023

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Operator

Thank you for joining BioMarin Second Quarterly Results Conference Call. Hosting today's call from BioMarin is Traci McCarty, Head of Investor Relations at BioMarin. Please go ahead, Traci.

Traci McCarty

Thank you, Katie, and thank you, everyone for joining us today. To remind you, this nonconfidential 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 authority, the market materially programs, BioMarin's actions with differ those XX-K pharmaceutical of and and Exchange actions availability and as the may Securities on in Commission, of the product in factors and Results detailed competitors such BioMarin's XX-Q, by of reports. capital, X-K

Commercial Chief team Officer; Executive Research Greg from Executive call Officer; Financial JJ are Chief the President Worldwide today and Fuchs, Officer. Brian and BioMarin's Vice Hank management Chief Jeff President, and Officer; Guyer, Chairman President, On Ajer, Executive Mueller, Executive and Chief President Bienaime, Development; Vice Vice Technical

to BioMarin's call I will CEO, the now Chairman our turn JJ over and Bienaime.

Jean-Jacques Bienaime

you call. in around on anticipated us Thank you, of VOXZOGO for more quarter access Traci, joining to ROCTAVIAN We was our the progress very and and this pleased the with second world gain families as highly Thank today the good approval were everyone. FDA received. afternoon,

of revenues these the growth Record growth expansion execution. launch milestones of represented billion of growth the year-over-year XX% and KUVAN. year excluding ROCTAVIAN first continued our XX% key plans, in half including components approximately and VOXZOGO achievement $X.X of The are two

this commitment net $XX were million strong Our income of quarter in as demonstrated profitability to pleased was demand. again product GAAP we and to deliver a result

at coming in VOXZOGO including $XXX million, revenues $XXX total QX With in revenues. million

as more objectives XX% communicated achieving XXXX XXXX is to a of revenue line drive are We leverage. path year. bottom this profitability growth significant than our This growth double-digit on expected earlier to in in and operating

and one-time we quarter, for clinical of treatment. for benefit the of very living transformative hemophilia pleased were to We has therapy demonstrated approval A received severe FDA's with believe gene those potential eligible ROCTAVIAN the in as have this

following chronic optimistic therapy. encouraged seeking signals we combined will XXXX to European team moved quickly have for an States. that The of of the choice those population first approval and are by addressable of in in treatment the With patients, of and alternative to US early interest activate FDA's the ROCTAVIAN we launch ROCTAVIAN the patient nearly commercial become phase United been

Germany and have will countries progress European provide in more made Jeff good the in a quarter, other launch during and We details also moment.

Turning VOXZOGO, cadence impressed to we uptake continue to be of by worldwide. the

of a and full year VOXZOGO initial guidance As significant we our this $XXX raising the million $XXX to a February from representing strong for and second continued $XXX result of million. $XXX between increase are between demand, year million time million, in guidance of

provide achondroplasia, guidance demand Building Jeff are for on treatment detail begin VOXZOGO our the will additional our in we treatment for hypochondroplasia. of significant VOXZOGO with today announce of plans the to to moment. on pivotal program pleased a VOXZOGO for the

plans program years safety we expansion fast seven to our as of achondroplasia, With and a track clinical over patient our provide data are highly suffice new population. it nearly efficacy view in VOXZOGO will potential more details we two rapidly from commercial and are years of VOXZOGO. executing the underserved in and in on moment. de-risked Hank say, second a But on experience

it drive that In we quarter expansion, product. first second date. for blockbuster potentially the will to BioMarin's The pleased summary, very our margin year continues VOXZOGO our confidence global performance we be supporting are and with to demand revenue in will record be and

the over awareness to meaningful result hemophilia will both patients through and excited States ahead United community ROCTAVIAN, the our direct the and about physicians the of A the Europe. We quarters. outreach ability execution on raise believe opportunity remain demand in in among ROCTAVIAN With launch US we're and focused coming in to

business commercial over call support. for continued now discuss I your to And will the -- the turn Jeff? you update. Jeff now Thank to

Jeffrey Ajer

KUVAN I'm JJ. in XX% pleased growth, total in year-over-year, with very and revenues $XXX including KUVAN. in quarter, XX% representing commercial growth million our you, second Thank and the resulting excluding performance

year Contributions well from us significant our to enzyme as products this full to XXXX deliver XXXX on BioMarin's provide revenues. as franchise, for guidance quarter the in contributions total keep full track year

Turning to VOXZOGO. continues our Today, year penetration expectations. to new as we patient again exceed increased full aggressive guidance

As midpoint noted to and at year raised today, of release in revenues the guidance. growth again representing press we guidance year-over-year the VOXZOGO $XXX, $XXX full XXX% once between

year on are run of $XXX managing based half Appreciating VOXZOGO the of note that that into may year. tight supply seem the we the temporarily first revenues XXXX. the remainder for rate million the of in muted We

new our VOXZOGO. achieve patients of to are inventory growth, managing closely limiting stocking main is goal, to which maintain continuity we including We that ensure that ensure

we while our are new the and hundreds focusing patient Importantly, patient levels starts inventory of new for supply in half of on plan provides this second starts, year.

future hand indications. possible drug do achondroplasia we on demands manufacture to Novato We capacity other sufficient have substance ample all and which have facility our for meet and at

capacity the our additional CMO, which constrained secured have element at is process. the We of Fill/Finish

were ensure beyond These million with the exceed currently full in will the second of XXXX end on XXXX. treated XXXX $XXX more accelerated At steps VOXZOGO. consensus, achondroplasia year and than to FactSet quarter, have ample supply different that being children at XX we support with markets growth

footprint, of XX% remaining. to patients the Uptake growth penetration represents potential in highlighting indicated commercial BioMarin's significant date

FDA very been receiving progress since We've pleased XXth. ROCTAVIAN. with to US on June approval launch now Turning

at the the centers value US. facilitate Following campaign in-process the payers the we've approval, educating the inflow uptake that been number and the ROCTAVIAN including of treatment centers of the pleased purchasing on team weeks or since In the patients, treatment across we our will contract agreement and ROCTAVIAN. HTC stakeholders approval, hemophilia immediately access patient with agreements, expect of the warranty consent and four or of hemophilia utility executed forms, executed outreach began increasing of

facilitate commercial to are ROCTAVIAN. access There many to activities ongoing

process centers the most on-site largest, with currently readiness. hemophilia are in We of capable working treatment

the with engaging has actively and payers On market been to the policies. patient access US facilitate access our coverage payer of side, team issuance

by process. issued US, being policies the ROCTAVIAN through get is coverage and exception commercial medical approval patients in once have individual available of payers for the we ability to the advance An

ROCTAVIAN US in are we to hemophilia key actively SalesForce promoting and the the in been community all summary, In regions. activated has the

We expect be forward ROCTAVIAN to of in available updating and coming to months. progress you pharmacies to look the over our labeled to ship August commercial

of As a of could with we on stated on to our approval scheduled June the location, we take the months two complete ROCTAVIAN HTCs insurer treatment expect from and with necessary call, eligibility visits to before it depending five testing. cadence completion regularly patient's steps

Europe, good Moving make we to briefly updates ROCTAVIAN to progress. continue in and

Germany. In

funnel resulting treatment with potential tests preparing robust in antibody in for continue of Germany AAVX patients come New to Octavia. through, a

negotiations reimbursement federal treated patient authorizations pricing final for insurers. While named possible with under are individual ongoing, patients Octavia is through

price has sales we been once would established. are and making progress. good France, to be Italy final In Those subject also it the

launch moving Our conclude as negotiations ahead where countries in activities QX application seeking by to are as approvals both we of price other preparation well and reimbursement expect year. this

continue interest early other and the to Argentina we We ROCTAVIAN including by Saudi authorizations access through have where in encouraged be named potential Arabia, markets, to provide patient and ROCTAVIAN to

which maintain the $XX full in progress year with we XXXX guidance. our we year are arrangements providing training between support We pioneering as we follow current novel required has million. Appreciating those of million of outside effort markets start seeing of ROCTAVIAN for reimbursement pleased guidance that $XXX States. and on full significant XXXX United the launching as well to together, at are to the Briefly a are up. ROCTAVIAN therapy treatment Taken truly

the several believe there outcomes months. potential over We are next of a variety

The supported one-time inflow by forms range for guidance is confidence the both Requests treatment globally and ongoing reimbursement. the warranties feedback patient Our the of from physicians ROCTAVIAN in of consent and patients from in and Europe. as nature testing and well treatment. in progress as US CDx

slightly For XX the perspective, guidance. US midpoint current patients of more achieved the of treatment than

In So global as stay to total at we midpoint coming track revenue XXXX, track over of progress. are months, we on full-year the conclusion, achieve tuned the guides. launch

unique profile far of to the VOXZOGO opportunity a this our of contributing in reimbursement marketed of half program during the fiscal hypochondroplasia possibility treatment for of second year, new the resulting two indication to The product. opens guidance start lends VOXZOGO treatment one-time itself the of for a meaningfully so year. increases with ROCTAVIAN expectations, exceed continues the new and

now of billion growing. are that base delivers and nearly established on opportunities annually $X business BioMarin's These layered top

this Taken earlier together, goals we are well profitability year. positioned to outlined financial growth achieve and

Thank update. you R&D And call now attention. the your Hank? an will for over I turn to provide to Hank

Henry Fuchs

Echoing Food and Jeff, Thanks, A in access and with States that approval the all enthusiasm to of are the extremely innovative Drug and joining therapy. thank XX gratified Jeff's June have Administration hemophilia severe this ROCTAVIAN, us for for we people living you today. United JJ

believe for achieves ROCTAVIAN interventions is with goal genetic BioMarin's Our goal. therapeutic conditions, we improving people outcomes with each and that health of clearly

unmet potential variety have conditions, of we has of genetic believe As represent we a VOXZOGO previously, the significant treat need. statural stated many to which

plans mentioned, elucidated approximately we global of VOXZOGO pivotal of XX,XXX have disease spectrum and begin across program with to the the JJ the in BioMarin's for be treatment individuals the be territories patient estimate course hypochondroplasia. of As population We study. the expect our full to solidified over with hypochondroplasia to the

stature feet. a is genetic by characterized dysplasia reminder, hands a and short As arms, small skeletal hypochondroplasia and legs, disproportionately

patients one us, with the a had of Dauber, is for genetic tool statural run of not multitude Andrew in BioMarin work study who of Dr. skeletal diseases, II improve doctors educated and outcomes does VOXZOGO deficiency. well has to hormone to growth As only outside in supporting Phase conditions it decades,

severe forward in improving achondroplasia. BioMarin, children with health to now ushering era impairment outcomes is new a look and growth for typical in beyond we As in

based from with new design on to the VOXZOGO we and indications. these in study on interactions the Dauber's our set emerging aligned a study, Following test FDA data Dr.

VOXZOGO directly in of in are regulator six-month our be later observation hypochondroplasia. pleased to trial. study later the a natural profile randomized, to growth, double-blind, of arm the by achondroplasia XX-week into we the development as followed a plan and study clinical by extensive this phase We clinical moving participant begin of the Supported placebo-controlled year, a of of pivotal XX -- bone program program

on interesting potential XX. get of we across on in earlier-stage treatment programs option for informative excited the for and Based we've our September the an been with the Day our for update enthusiasm we're to hypochydroplasia. on R&D VOXZOGO the Briefly, first started children treatment have pipeline upcoming seen working with on achondroplasia, together pipeline,

product for The in disclosed for updates. publicly protein XXX hyperoxaluria deficiency, cardiomyopathy. hereditary our disease, BMN well and CX deficiency includes as BMN BMN XXX gene muscular new angioedema, five for Duchenne other chronic agenda for liver as candidates alpha-X myosin-binding BMN hypertrophic for XXX antitrypsin includes causing XXX BMN dystrophy XXX This therapy

happening been areas part to interesting potential of an and the key focus commercial pipeline, the for BioMarin hope an you'll leaders next chats Day. update in on product important sharing And of what's look that to be our candidates. tune therapeutic key some about R&D learn in fireside forward with or opinion We earlier-stage we and will more

And call Thanks in for Brian. Over results the to support. Brian financial I'll to over update to turn your the you, now quarter.

Brian Mueller

today's of more revenue key XXXX. results details you, to revenue Please then comments, for summarizing we'll I'll Thank P&L focus press performance Since JJ on of our quarter spoke Hank. our outlook, quarter quarter. remainder make the on this to other and for just and revenue refer full second the updates Jeff future financial few financial the our release and a

will we couple of XX-Q, file results the days. our which on upcoming next usual, track be over all Form available As to in are

of of XXXX. previously VOXZOGO, full GAAP EU, of and milestone noted, foundation driven growth we important we BioMarin, the as As transformative year double-digit last in laying growth year profitability by the our and approval have year a ROCTAVIAN sustainable revenue our viewed of strategy

to performance the with the P&L build of we continued on first U.S. in that ROCTAVIAN strong that As growth we're aligns our the approval foundation and long-term half half revenue financial and to pleased of close first of objectives leverage. XXXX,

total of in XXXX of second million of quarter $XXX the second to of the is XX% quarter an revenue BioMarin's XXXX. increase compared

midpoint double-digit of revenue outlook, reaffirmed revenue continue our of growth XX%, total at to for the the rest Regarding anticipate strong guidance. our we of XXXX,

given is in half million, first guidance performance come in that over earlier, has the Jeff VOXZOGO. with the end pleased low appropriate to revenue note our $XXX And prior felt we mentioned line of it are our first longer no As we expectations. of half with our

million. So guidance $XXX year, an that provided to for million mind, slightly we the to view with have updated $XXX raising in

the Moving second was margin XX.X%, X.X% XXXX. improvement which to as an is past revenue. QX of gross compared XXXX of quarter

performance the with XXXX improve are and We half of our first metric fundamental it product through pleased mix. our as gross over favorable objective reflects cost margin efficiencies this to

revenue. XX% million and with than base grow $XXX expense $XXX million expense of and XXXX QX versus QX grew goal line respectively, X% slower and in in of expense R&D SG&A our to XXXX,

While full half basis today we that we progress study the to the continue as Phase new we for do a and hypochondroplasia, year of R&D growth ROCTAVIAN to to in expense expect align including on investments pipeline, expenses III a second goal, acceleration operating anticipate our launches. announced VOXZOGO and

we QX of profitability This line, of bottom million achieve XXXX our deliver with and to sustained of full in $XX the continue objective On to profitability income. year on million stated income non-GAAP our GAAP positions net $XXX commitment us and GAAP to going forward. growing

our respectively. continued XXXX. performance updated income adjustment, GAAP to $XXX to $XXX in our million updated guidance revenue to for we XX% income $XXX more reflects VOXZOGO of the XXXX strong meaningful our represents guidance our midpoint, to $XXX goal. which financial non-GAAP growth, expectations bottom revenue year Today, the million transformation and and expect we with also in million, first is outlook million revenue line and the in growth Similar half half and At back the leverage versus net of line the than our of

recent to cash over on to enable next place, profitability beyond. track our years. VOXZOGO launch, for and of revenue coupled US, with ROCTAVIAN ability The course in closing, flows the strong further the will objectives of meet in successful expand drive meaningful approval the we In foundation are the growth, and generate the several our including XXXX

open attention. Operator? your call up Thank And to questions. you for now your we'll the

Operator

Tewari now session. Instructions] question-and-answer [Operator will conduct Akash Jefferies. the comes question Thank a and we from first of Your gentlemen, from you. line Ladies

Your line open. is now

Akash Tewari

Hey, thanks so much.

the like commercial But XXX product won't ROCTAVIAN going of update excellence. actually in diagnostic some centers August. actually interactions on you've who've patients the or leading sounds been the companion of you center until a patients with in had I previously into any just indicators with mentioned to think you're gone So previously you've late US? get see to distribution have terms regarding treated the It treatment

also Should would number Phase So number. for data at data. they did FDA guess, two, time a on any for that wouldn't years require on like hyperchondroplasia? I'd update two X love I lines VOXZOGO on then, what data why hypochondroplasia you comment the And bit in And dose syndrome, need Phase we there? maybe Can right? about worth be five to that worth you you would for right I Noonan expect XX patients' have about Thank X think XX-microgram Noonan in data supported patients' approval of of now. be the you. of

Jean-Jacques Bienaime

and Jeff, the want question you Hank? do to with first start then

Jeffrey Ajer

with Hi Yes. with I'll the with launch US question. first qualified estimated case patient Akash, It's XXX absolutely the started start an leads. we your that

further and of been one with into in are an these had individuals had opted our touch through properties digital So that either in-person engagement BioMarin information. or

assure noted, As those team to has I to been as expect a are can place noted XXX call actively. back and processing falling that fact And through that you those signals ago, of our in on our sales getting last approval patient And for coming list top in month into engagements ways, and digitally and the in. demand priority we of different force. lead is pieces you seeing month in small consent responses from of to forms the leads

Noonan and with over So it things And maybe Hank to that on are questions. on encouraging I'd really hyperchon that, turn front.

Henry Fuchs

Yes. the Thanks, Jeff, inquiry. and thanks, Akash, for

study a time as registration. driven the and the across Noonan, being FDA of a to of a of very by Voxzogo further bone has in VOXZOGO least achondroplasia at does that eligibility, good baseline, that hypochondroplasia, their Dr. I is is their designs, abnormalities hypochondroplasia think hypochondroplasia. IST we those question they're that with for you endpoints up safety in of borne really is terms at just skeletal health analog And -- as in regulator, And done dysplasias. communicate comparators, skeletal two growth. VOXZOGO in as years lines and the given study duration syndrome growth our anticipated He's development with We're beyond contrast more durability This discussions on its And record support future exciting disproportionate out as with data maintained variety that sense plans achondroplasia are for out FDA bone growth finalize data, and its great velocity safety being Noonans sufficient natural for and now effective noted. Noonan be for really on regards level interested could a your In of Dauber's it -- activity durations. mutations, been with that's pin anticipated potential specifically bone study evaluating regulator range indications, And in patients comfort least lined make we a natural treated the the the down three it above we've of importantly, the and But VOXZOGO collected points would others. as patients of of a in year. gaining Noonans. regulator an of and to is be for in we'll authorities based have one-year which other

Traci McCarty

please. Next question

Operator

from line Your Salveen next Sachs. the question comes Richter from of Goldman

up. line Your now is

Salveen Richter

you. Can afternoon. for ROCTAVIAN and the the help the to second half of in you context in understand EU confidence the guidance us US dynamics? Good Thank

Jean-Jacques Bienaime

start -- and have I'll I'll Maybe Jeff going this. let I'll

price $X guidance be prepared in I million, ROCTAVIAN think based as US, to would million Jeff midpoint is the US $XXX of the of remarks, which said our patients. the on XX in net close the only

therapy the instance, terms chronic year chronic or is also early in something revenue year recognition late therapy. the patients. special the forecast difference the to in in in of VOXZOGO, for VOXZOGO as patient therapy because makes because US And a that ex We gene non-US, that's of are about counting any just compared starting

patient if you the to if same treat If the August revenue. start December revenues of we a than will this XXth, December Xst. February for But XXth, much you smaller the -- start a on it's ROCTAVIAN, year Xst, on in on them or be on atient

Jeff? So this introduction, just mind. in With keep that

Jeffrey Ajer

Yes. Thanks, JJ.

think I And thanks that's the exactly for right. question, Salveen.

of other in Germany, that revenue. seeing now mentioned are add France, which saying XX that of open things weeks I've pathways and of for from couple we for to typically up we're to I The I price Italy progress would all expect occasions ROCTAVIAN multiple year are XX process. European priority to markets since reimbursement. numerous away just on We're that get been a now approval. takes Remember, and quoted X through months couple

should approval be named end quietly the has getting and patients we after the availability those product the where we a but Saudi patient or So now we've of end patients. Also in point the for markets for opening United up Argentina on all been the and the can at to quoted, I've of months the working in year, process But pretty background. Arabia and our been start-up approval. that treated we treating that the for pathway quickly midyear between markets a of some finally, generally And got in takes get couple our experience US, the States of diligently activities.

channels and of able things the up add all in add I guidance to that those treat of range. revenue to for confidence being So think patients being to

Salveen Richter

Thank you.

Operator

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

open. Your now line is

Geoffrey Meacham

the for Thanks guys. Hey, question.

Just few. a had

rolled base. was driven. is, Hank, I you I of commercially, commercially Are know a in it's in metrics expanding tweaked centers been the if you new just then since achondroplasia the get Dauber's mechanism-driven wasn't patients? since of sort have months strategy to hypochondroplasia you diagnostic can globally a in VOXZOGO, us, you. you've the in you strategy. work Dr. progress out the or there talked, volumes? ROCTAVIAN I And access the few for shift give want either as Europe, about X identified Germany. activated had guess you question reimbursement sense any for sure indication on I or a Thank Phase kind So the

Jeffrey Ajer

I'll maybe ROCTAVIAN. Geoff, Germany the and Hi, with about question start

funnel robust CDx eligibility testing quoted are a testing JJ reimbursement health approvals. As got going patients patients call, really the minutes, through go process for liver via we've eligible actually now prescription approval through process, and a of that testing on CDx, our in in following

really quoted about last fall, Germany. So JJ with funnel -- adopter XX early that XX healthy patient view I patients, we ago, as a Recall in a estimated as patients starting. said month approximately we

they're at and frustrating the it's formal number, we the we're particularly before I'll on Those And to there, that's confident patients, progress with as So making through to Hank that reimbursement. understand the of question. reimbursement that agreement the we see revenue I that the I'm other federal process. X.Xx VOXZOGO making we'll out that if authorities are and popping over Maybe price them side patients and federal treated can but inside price come get not an get and to on turn progress funnel.

Henry Fuchs

that mechanistic themselves to and other from is a growth. a of indications. patients as lead conditions the traction, similarity. available very abnormalities And many is achondroplasia Dr. into more Thanks, the be clinicians or are are point in centers getting our now And much of further tuned in And treatment, disorders for to hypochondroplasia. a time. and the call, achondroplasia, no a identifying CNP with by to will said gaining identifying think so on Jeff. that updates suffering genetic of study. no strategy extend forwarded identification, regulatory diagnosis. And It's enable this to families process skeletal to that for study, and I about hypochondroplasia, I ROCTAVIAN bone And be skeletal a exciting us Dauber's beyond that -- other natural stay more families children I similarity begin than VOXZOGO additional to there With this lot into could the regulator expectation will as themselves of is think as case is hypochondroplasia recognize when the to for patients your with his with achondroplasia phenotypic there of driven tends lot referral with themselves hypochondroplasia treatment

Geoffrey Meacham

you. Thank Great.

Operator

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

is line now Your open.

Christopher Raymond

thanks. Hey,

of questions. couple a Just

VOXZOGO First on bit. the issue, maybe little I a expand you Jeff, wonder supply if could

raised but heard handle And think 'XX what have fill-finish to full I on expand more. supply say I But brackets XXXX you exactly is plan longer you action can at said a just the issue wasn't no could what's timing put year so some Like an the to It's supply around you supply-constrained? I this? the maybe guidance a maybe it's consensus, CMO. for sort the you with to pipeline. think and And planned issue? of the be why you maybe then Like situation bottleneck? it's not deal Hank on the

can Just on this into sort for on looking Thanks. now there that. XX, is program expression any or talk two the a -- the color yet verbiage X the Maybe got decision not drive about would what at of you've go/no-go just therapeutic range. patients trajectory on where more you

Jean-Jacques Bienaime

Thanks, Chris.

We have answer new to Greg your Guyer, our supply Head operations VOXZOGO of who's taking question. going here,

Greg Guyer

and a that just That we're remains just And maybe about clarify consensus plan perspective. a that for to Jeff was even patients mentioned about from the said, an hundreds earlier with supply that exceeds second that this year currently, question. supply of in many bit FactSet. tight half the estimates he supply XXXX inventory little of starts have is in what thanks the able to talking Chris, Yes, VOXZOGO

reliable, to continue they're we a partner, So They're accelerate supply we this My to CMO great to just team just which to the everything escalate dependable, use. from high it group. appropriate can or we escalate is thought compliance. it doing was

to So than of supply faster a to the getting much we type there's accelerate It's rates And expected. commercial planned. for had no we than trying availability really issue. tribute really what penetration our that's faster just the had team

closely make on term. very to we meet that also working demand that are of sure deliver them and we potential future long VOXZOGO can full so And with the

Jean-Jacques Bienaime

ourselves in there's details is substance, manufacture supplier. drug no issue. a the manufacturing -- manufacturing here in done again, terms the of by fill-finish in California. The There we is outside So

'XX to terms when increase up of 'XX. a have 'XX, And we capacity. what's But we're I in volume. I suppliers, you it's clearly shouldn't time is what capacity But drug billion. a factor. can down ease the if of any a lead the way, As in little have limiting anybody, $X unlimited, way get than creating because -- anticipated if we further. the substance going mean here but by end know, way we including it's successful, is that's more really billion and can unlimited would we you doing want we go VOXZOGO outside to -- lot even is facing better again, yourself, $X to have at That's penetration. then of And believe we beyond, Greg say above in nothing drug substance hypochon almost go we and And need problem. -- probably or road, there's

do next to our that that same problem in And increased we higher, current that 'XX us for But actually, including fill-finish that allow believe be the consensus would above to want in So Jeff, the easily -- will for today. are it guidance correct can have did if bottleneck, communicated have year, the you're good we news just your at anything? have remark and we is that supplier have. should what But the we temporary to in -- feels our VOXZOGO front, add it much fill-finish is plan, bottleneck, what easier. XXXX. which we time, beat do we -- you

Jeffrey Ajer

stated. No, you. all Thank

Jean-Jacques Bienaime

Is there Hank? any comment, Yes?

Henry Fuchs

good One could a be be yes, before criteria would pretty at safety XXX, taken note for stage. the to clear reaching profile. option On very this raise and that therapies staffing, Chris, do we top criteria have have those Yes. dose. AAVX-based

we contemplate so the dose. could raising And

very patients bit in And be the not have we're Another new much therapy. Data Monitoring more we would resume to the thing some if I'm I'll a we'll criteria. but provide data with optimizing -- further yet, course, -- successful, Committee very we program. with study with a treated just are insights working as it's those And obtain gene and as and closely little meet those going mention have, and we expression the early stop is as And updates. new gene of dosing about neither we for that insights. But investigators specific about this ROCTAVIAN data, we two of of to incorporate still

Christopher Raymond

Thank you.

Operator

Your next from the of question from comes Securities. Truist Karnauskas Robyn line

now. is line Your

Robyn Karnauskas

Hi. Thanks. I'll be quick.

insight is we long-term follow-up. And landscape I Can Number you. the do Thank two, that many have have you questions. do committed one into you elaborate? hypochondroplasia. studies? questions to one, three -- Number to how and compliance high interns as Any around then So competitive VOXZOGO. studies there's very in gets that? Like are. heard And

Jeffrey Ajer

Maybe start compliance Robyn. with as on it's VOXZOGO, with very note, And high. a you question

We implicitly in measure able to are all not markets. that

market one can the in the be the that experience high. So daily dial we so as the measure and on very in to is States. few States appears with that United it drop-offs dosing is And very United our far really compliance we

So, so maybe far, And it over so I'll compliance side. the to good turn Hank. on

Henry Fuchs

one growing which we've with Yes, thanks, Jeff. interacted placebo-controlled requirements would study. clear with regards hypochondroplasia requiring the satisfied study question, and In for with of to be longer-term And Food randomized satisfaction regard not Administration the pretty as a durability. their period have is picture one the and reason Drug their follow-up

further is little achondroplasia required even where for at a of no In hypochondroplasia, to depending further, time even regulatory fact, maybe be may on the action the hypochondroplasia. go on follow-up longer

hypochondroplasia as to not data, demand supplemental in excellent for record are applications dialogue as with there's necessarily new drug we a great excellent with track in the patients subject with So accumulating an so of dose. same these the initial safety And FDA. had sorts VOXZOGO the far efficacy

you pretty So And you mentioned had a question? excited. follow-up

Robyn Karnauskas

sorry. follow-up, One Yes.

was letting expectation about patients curious, us some during thought like much so or There's you'll Analyst fixation you there's dose the Europe. So on like have on I know that. in earlier Day? And when just

on us color give clarity much give maybe might timing see that excitement, we're yet So of to the when there's and you dosing. but because would us how give you

Jean-Jacques Bienaime

but a I your patient, that that, one call on announced with scheduled Actually, physician Robyn, to German financial recent revision analyst That XXth on -- August Yes. treat one competitor call, German of think, first or XXst. his a is physician. had that

that patient possibility the some given. last one [indiscernible]. we should this after also start getting would on to And treat September a going So be And is rest of the probably August our we're revenues. that traction looks first then and treatments real patients end like when then really by

Robyn Karnauskas

Great. Thank you.

Operator

TD Your next from line from Phil Nadeau of comes the question Cowen.

is now open. line Your

Phil Nadeau

consent taking our to forms on that well. a consent knew guidance. over afternoon. your Is form the the last JJ, questions as for approval? as you patients bit comment? as the month? in you the during confidence on of prepared go you more ROCTAVIAN. adding Couple US there more of remarks, Good been Thanks into mentioned Can the simply time received XXX the have Or that incremental of detail that received

Jean-Jacques Bienaime

We received I'll let We -- I patients XXX already consent question. want that don't that. to believe Jeff answer form.

can We they're here. some provide in and haven't, -- Jeff but roll more but color maybe starting to

Jeffrey Ajer

Yes. Thanks for the question, Phil.

rep at patient, up So management not a of be for all example. the these to are we use lead. sets we're into case have vehicle are in independent qualified hand, lead forms, information. competitor of further a XXX hemophilia following we opted ROCTAVIAN to means patients. leads United the that our for that qualified are sales intake those a our And a The really patients have patients for conduct Patient quoted States, other company, usual we've system. patient programs, including in We've on for that consent

be any with form for treatment And might come system along consent rep and as the with case, independently touch that with we from patient coming them a in been patient-directed in sales patient or connect be management patient center coming consent prescription interest. a for follow-up. would depending, in hemophilia in in our has that for opted case services, has with in further that's So might

coming good all mix, it's we it's really of really, a that have programs, including forms consent system. our patient how but Roctavian. management it us with into always begins for So case our That's

Phil Nadeau

consent to you care you have? forms many disclose how Would patient

Jeffrey Ajer

Thanks at for this Not time. effort, the though.

Phil Nadeau

make to a patients? you in therapy? do have permission can ROCTAVIAN a has -- is its center accept committee then formulary or Any do -- bring committees ROCTAVIAN a they order patient? to ROCTAVIAN from to before to there for other to that of they there as the And a need In get does it. Got themselves, that terms talk available centers do general, in what to

Jeffrey Ajer

description. of the site no It's notion a there's important one-size-fits-all readiness. and unfortunately, question, an of great simple And kind

or health HTC Some do centers that that and those you and procedures treatment into hemophilia maybe HTCs system. care all own. the a In from you also connected more of for cases, formulary, the cases, other an standalone larger tap larger services their to might example, on less like that are have And administrative mentioned, are pharmacy in institution.

with larger capability HTC readiness the connected depending is size site different on the are and institution. they not or So care a health looks what of

in administration, of trivial about value, and Just the call. of frozen we reminder, patient a follow-up ROCTAVIAN so, if interactions, interest to on example, example, important them and with counseling a a are determine administration, patient the the for testing product is a relatively handling which product high there's issues the eligibility like a the moving navigating very has things process But that then if forward approval for formulary, more talked infusion and following payer step. the

going those on -- each it. a list rather done big bunch of smaller on lots whole them team It's hemophilia there. None our hurdle. is things be of that that to need things and bespoke for and a treatment involve for mostly So of is center, a

Phil Nadeau

can that Great. you follow-up enzyme patients? for you onerous requirements? And something the very Is easy last then are monitoring and the that that that on mentioned, liver just factor how make

Jeffrey Ajer

levels. that, us expression are the tests or a time same that Blood test. panel of is a year. a can simple taken. being test be blood function most test. simple once At blood do used that That for Liver on It's probably factor blood twice

Jean-Jacques Bienaime

to that? patients you -- The do Do want

Jeffrey Ajer

That's right.

of In a going the have a patients, it's nearby we assist. simple to very as lab convenient for facility. that, have as programs testing patients So to to access don't that lot of case

Phil Nadeau

for Thanks taking our Perfect. questions.

Operator

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

line Your now is open.

Joseph Schwartz

much. so Thanks Great.

many indicate uncertainty Our be which will about checks how some and there. still that Germany sites there is in able ROCTAVIAN to administer

expected it wondering you're process talk So up reimbursement this and this was I which expect works about you of to play part follow-up done Or this initial be on underscore checks that the rule-making reliably. something out. could the you contracting our if U.S., wrap to Is negotiations in longer-term the important the separate? how how September? sites is And then with can patient in performance ordering the and

question, doing So I U.S. Thank HTCs can in insight of been any to follow-on that into wondering contracting you. sort Phil's was of you've if the us with give the you extent a

Jeffrey Ajer

Joe. Hi,

start me with Germany. Let

the a even over Europe, model hub-and-spoke have been that's not markets for year that. And behind the community is BioMarin driver about major or longer heard you've talk So around. me hemophilia last the in Europe the treatment rallying in treatment

be infusions of list treatment the fact, probably a spoke all So infusions capable that likely for the matter centers screening entire treatment. which spoke of the follow-up the the we're with of then hub on center from hub would Germany. do promotional to and refer very smaller, of a there treatment screening and all little the centers centers for of And the would those treatment who's perspective, hub hemophilia in at focused most be would be spoke. done on from a center both largest that, centers in less recommending Germany, it the and and But perspective which who's There of would testing centers doesn't bit would for centers. really spoke uncertainty The treatment. on be back example, the treatment significant kind much engaging notion practical And the for Germany. hemophilia and centers in and be is a that centers capable after and hemophilia do may, patients

at to the with on United of might you're agreement desire contract on a not treatment might sites to center certainly and be for a which to ROCTAVIAN, United spoke center contracting be a all of or for and need States, likely there part further if in between level a between spoke what sites sites, a patients and treatment a one contracting talking analogy, an their hemophilia that's hub think treatment not involve reached would You hemophilia There mentioned a contracting, payer. refer kind patient-specific barrier readiness to treatment. one the capable a along and of States, and more the of use We ROCTAVIAN. I'm center in payer reimbursement of between that's the to there's sure site exactly about. a treatment the also the that big proceed center In

any haven't are to proceeding are to of there contracting. All things those possible. that I barriers heard personally due

got you've specific, something If more stock.

Joseph Schwartz

good. No, that's

Jean-Jacques Bienaime

the Basically, treatment some basically gave chemo with ROCTAVIAN discounts, Sorry, treatment are U.S. I of be treated XXXB aisles. centers. as for centers may the in receiving that investor patients hemophilia Joe, are our add, statutory all will in centers

more take That's and than But go it WACC be Let's to will whatever. XX% to around -- it initially might the XX%. cost. it let's XX%, round XX.X%, down of

So WACC patient. million a $X a is

of go HTCs per $XXX,XXX the patient. is in will So the XX% pocket that

might or this you you -- information this might interesting. have So information find

Joseph Schwartz

Very so. again. Thanks much

Operator

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

Your line is now open.

Paul Matteis

terms you and Hey, timing, before it when the supply why this much impact done your in get with that sort took to seven you did? think the in reasons might you approval, I you to just of health just that the temporarily the seek Hemgenix think than months And thanks way dose guess, CSL X-month how the mind providers how so taking if does couple you X- issue ROCTAVIAN to of expanded can the so timing much the Can On patients issue is or, Jeff, faster around and key for to question. first of on care resolved. fully capacity label capacity constraint the market VOXZOGO, around patient. for then impact my the drug? spoke interact is outline I And you

Jean-Jacques Bienaime

in prepared our, Jeff current on our the can will said XXXX, which then good remarks $XXX that we ample consensus, Again, supply chip to have the I'll and is VOXZOGO beat and million. question. -- to close we start Yes, when in. for

that us, potential expansion includes increase in mean for So -- of terms patients. label a of new I

are some. patients under New AAVX, occur, that if does

that's So included. already

anything have do in add the you share doesn't here? respect. anything that made I this we Greg, to it mean, So remarks in

Greg Guyer

hypochon No. be not supply And one of by have will this plenty issue we'll the supply. time -- comes,

Jean-Jacques Bienaime

-- hypochon hypochon not we're condition the Yes, going next get year. to

Paul Matteis

okay, younger but meant patients, thanks. Just

Jean-Jacques Bienaime

our for year supply but million. very significantly with current enough the And we guidance significantly is consensus XXXX Younger of Jeff, are this have patients, the -- patients you of consensus, anything top whatever add want to guess, already, to the I the current do to... beat $XXX above we comfortable are.

Jeffrey Ajer

patients to X,XXX through it into metrics we world; the of Maybe QX, guided of supply QX, plan there therapy. additional around X,XXX on advised put patients And were a this Yes. couple perspective. treatment end end year just that of the for of world. At around our allows end we've the a quarter-to-quarter. hundreds patients to the That's gain just of on that increase treatment big the

at overall this sooner in to. how big coming internally, is coming is not we're the surprise. just access demand So is faster uptake we're and It's looking the gaining markets a that

that want grow. were sooner, planning gaining on. access those to still markets So We we base faster need than in uptake and patient

And supply doesn't where And bumping keeping we're ceilings to rapidly good the of to certain highly that's in is some of started or just much go prioritizing, make fundamentally have for got that launch patients as our United treat in VOXZOGO. because into a alter On We're so ROCTAVIAN sure continuity kids for on -- what started really that we This happening, to ROCTAVIAN, That start, we've will really don't States us. motivated it for drug. off growth trajectory as supply that want have therapy. have this happen is we're places. kids possible.

have to with access. therapy this that cannibalize example, as quickly rapid hand, On are a as existing not been steps, the treatment we the But like of for hemophilia. new possible. steps number signed purchasing moving before of we entrant having an group need We a for day to it's for we took a We've contract we one we've approval. preparing our to gene got facilitate We're taken organization therapy. And even years. those

Jean-Jacques Bienaime

And warranty. the

Jeffrey Ajer

the got We've warranty.

consistent as manufacturer. differentiating taken the speak with for that as would be And the we've think so that So I moving can't rapidly fast. But I move desire are other far those steps factors. we're possible. to

Jean-Jacques Bienaime

intending warranty be that. And have presume there a payers Otherwise, that them they no are need cover means already will do signed contract. to several for to I VOXZOGO. That

Paul Matteis

you. Thank

Operator

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

Your line is now open.

Gena Wang

-- feedback just most comments. you question question your on ROCTAVIAN now payers, developing comment is warranty Germany you just are do you coverage hypochondroplasia. under XXXX expect since mentioned? centers from revenue one follow Thank Second program what right the questions. many in regarding JJ, the is also U. Maybe And place? and first, for my the Paul's And Maybe or for revenue, and lives VOXZOGO how regarding in US.?

help what trial? So Dr. is would regarding define from assumption? to for a And the be XX-week Dauber's learned actually the how what study, the you looking six-month you pivotal pivotal for study, the observation study? much trial And

Jeffrey Ajer

and Maybe I'll fill JJ let in. I'll Gena, start,

And in the that opportunities of we've for give even start those approaching sales rapid the from get States, would in described the treated price to with Europe, almost expect getting questions the of months now initial processes, for to Europe. in patient we period what would markets, expectations fact to confidence that and when that patients possibility price but our on the what a other at we're is and in major to that revenue markets I've noted, you United the the the formal conditional different as of think able named be a guidance and That we couple of where, through time contribute XX question, where finish for named is in we did received is reimbursement U.S. channels we States. part together only Salveen's United the approval least terms in In in revenue. I of not ROCTAVIAN occasions approval

the take a which of indicate those not yet be policies that language would both I've that In from United a label consistent lives, months X have we policies I'm trial clinical with coverage at have issue. least draft States seen policies coverage terms and -- coverage can that be coverage actually, to of been or don't to that will Those which would fine. either issued in percent anywhere around XX of. some aware policies issued yet, inclusion criteria,

haven't yet. to start issued seen policies We those be coverage

hyperchon. percent can't And United over comment Voxzogo the the covered on I for it and of questions So lives what Hank in would States. to I maybe on turn

Henry Fuchs

the document study rate Yes, sixth is to randomization run-in prospective important prior Gena, into growth annualized to baseline study. month the

rate an for relatively baseline which changes of where of study, important than expecting talked interpret we that As smaller the things growth bit be is of in purposes. to observed is The able really velocity the and to growth that, effect. the is a as study Voxzogo I magnitude is randomization knowledge a disorder subsequent we XX about achondroplasia, participants, we before, one similar growth of is area that little in are in mentioned, important that

the can that XX of be baseline course, on minus value the population. depending But just what VOXZOGO that baseline in Of for to to you, is XX. AGV the XXX-patient run tuned in study P is achondroplasia, remind in

Phase data the So all reassured anticipate achondroplasia. we're the about we from III quite has Dauber reassured a And to into as we the did evolving that seen we needing power clinical don't that Dr. in as trial. we've study been FDA go of based on can directly as aggressively this

thanks the for So questions.

Gena Wang

you. Thank

Operator

concludes conference That portion the of Q&A our call.

Bienaime, closing JJ to We will CEO, BioMarin's back turn it for remarks.

Jean-Jacques Bienaime

world in treatment physicians half to with Outstanding record of XXXX. coming you begin thank way in our Europe the led on to achondroplasia. our only business all months. are with US start around we Roctavian treating VOXZOGO on first And of and the the us the We more and across joining as well generic the revenues approved and operator, today. for in call you, Thank medicine the execution family starting reached with over the use patients children

the the and we the good profitability in build plan remainder have day. first for XXXX, a for to support, foundation achieved continued your of you of half Thank of and growth on year. So the

Operator

call. Ladies for and participation. gentlemen, this your you conference Thank concludes today's

You may now disconnect.

BioMarin Pharmaceutical (BMRN) 31 Jul 232023 Q2 Earnings call transcript