Exhibit 99.1
NEWS
Contacts:
For BioMarin: For Genzyme:
- ------------ -----------
Jeremy T. Price Dan Quinn (media)
Manager, Investor Relations (617) 591-5849
BioMarin Pharmaceutical Inc.
(415) 884-6777
Sharon Karlsberg Sally Curley (investors)
Vice President (617) 591-7140
Feinstein Kean Healthcare
(617) 577-8110
For Immediate Release:
BioMarin and Genzyme Initiate "Rolling" BLA Filing
For Approval to Market Aldurazyme in the United States
Novato, CA and Cambridge, MA, April 15, 2002 - BioMarin Pharmaceutical Inc.
(Nasdaq and Swiss SWX New Market: BMRN) and Genzyme General (Nasdaq: GENZ)
announced today that the companies have filed the first portion of a "rolling"
Biologics License Application (BLA) with the U.S. Food and Drug Administration
(FDA) for approval to market Aldurazyme(TM) (laronidase), an investigational
enzyme replacement therapy for patients with mucopolysaccharidosis I (MPS I), a
life-threatening genetic disease.
The companies plan to complete the BLA filing in the third quarter of this year.
The BLA will include six months of data from the ongoing open-label Phase 3
extension study in addition to the six-month data from the placebo-controlled
portion of the Phase 3 trial. Patients from both the treatment and placebo arms
of the Phase 3 trial had received at least six months of weekly Aldurazyme
infusions in the open-label extension study as of February 8, 2002. BioMarin and
Genzyme anticipate a response from the FDA regarding the application to market
Aldurazyme in the United States during the first half of 2003.
Following discussions with the FDA, BioMarin and Genzyme believe that including
longer-term clinical results from the extension study will provide additional
evidence in support of Aldurazyme's approval in the United States. The companies
continue to conduct a comprehensive clinical program to evaluate Aldurazyme's
potential benefit for MPS I patients.
On March 1, 2002, BioMarin and Genzyme submitted a Marketing Authorization
Application (MAA) to the European Agency for the Evaluation of Medicinal
Products (EMEA) for approval to market Aldurazyme in the European Union. The
Agency has accepted the MAA and validated that it is complete and ready for
scientific review.
Clinical Trial Update
BioMarin and Genzyme intend to initiate two new clinical studies in MPS I
patient populations not included in the Phase 3 trial. In one study, the
companies plan to enroll patients under five years old. In the other study, the
companies will seek to enroll patients with certain more advanced clinical
manifestations of MPS I. In addition, patients from the Phase 1 and Phase 3
open-label extension studies continue to receive treatment with Aldurazyme.
Both the U.S. and European marketing applications contain data from a Phase 3
clinical trial of Aldurazyme, from which preliminary results were reported last
November. That trial enrolled 45 patients at five sites in the United States,
Europe, and Canada. The randomized, double-blind, placebo-controlled study
showed a statistically significant increase in pulmonary capacity, as measured
by Forced Vital Capacity, and demonstrated a positive trend in endurance, as
measured by a six-minute walk test. The safety profile in the Phase 3 trial was
comparable between the treatment group and the placebo group, and there were no
Aldurazyme-related serious adverse events.
About MPS I
MPS I (also known as Hurler, Hurler-Scheie, and Scheie syndromes) is a
life-threatening genetic disease caused by a deficiency of the enzyme
alpha-L-iduronidase. The deficiency leads to the accumulation of complex
carbohydrates (GAGs) in the lysosomes of cells, leading to the progressive
dysfunction of cellular, tissue and organ systems. Resulting symptoms can
include impaired cardiac and pulmonary function, delayed physical development,
skeletal and joint deformities, reduced endurance, and in some cases, delayed
mental function. A majority of patients die before adulthood from complications
of the disease.
BioMarin and Genzyme General formed a joint venture in 1998 to develop and
commercialize Aldurazyme worldwide whereby BioMarin will manufacture Aldurazyme,
and Genzyme will have responsibility for the commercialization of the product.
The companies have received Orphan Drug designation and Fast Track status for
Aldurazyme for MPS I from the FDA and orphan medicinal product designation from
the EMEA.
Genzyme General develops and markets therapeutic products and diagnostic
products and services. Genzyme General has five therapeutic products on the
market and a strong pipeline of therapeutic products in development focused on
the treatment of genetic diseases and other chronic debilitating disorders with
well-defined patient populations. Genzyme General is a division of Genzyme Corp.
BioMarin Pharmaceutical Inc. specializes in the development and
commercialization of therapeutic enzyme products to treat serious,
life-threatening diseases and conditions.
This press release contains forward-looking statements, including without
limitation statements about: the expected timing of completion of the BLA filing
for Aldurazyme; the anticipated benefit of including data from the on-going
Phase 3 extension trial; the expected timing of FDA's response to the BLA; plans
for additional clinical studies; and the potential receipt of marketing
approvals for and launch of Aldurazyme. These statements are subject to risks
and uncertainties that could cause actual results to differ materially from
those projected in these forward-looking statements. These risks and
uncertainties include, among others: the results and timing of the on-going
Phase 3 trial; the actual timing and content of the completed BLA filing for
Aldurazyme; the content and timing of decisions made by the FDA and the EMEA
regarding Aldurazyme; the ability to manufacture sufficient quantities of
product for development and commercialization activities and to do so in a
timely manner; enrollment rates for clinical trials; the continued funding of
the joint venture between Genzyme and BioMarin; decisions made by physicians and
third party payers regarding the use of and reimbursement for Aldurazyme; our
ability to obtain and maintain adequate patent and other proprietary rights
protection for Aldurazyme; the scope, validity and enforceability of patents and
other proprietary rights held by third parties related to therapies for MPS I
and the actual impact of such patents and other rights on our ability to
commercialize Aldurazyme; the competitive environment for therapies for MPS I;
and the risks and uncertainties described in reports filed by Genzyme and
BioMarin with the Securities and Exchange Commission under the Securities
Exchange Act of 1934, as amended, including without limitation the factors
contained under the caption "Factors Affecting Future Operating Results" in
Exhibit 99.2 to Genzyme Corporation's 2001 Annual Report on Form 10-K. Genzyme
General Division common stock is a series of common stock of Genzyme
Corporation. Therefore, holders of Genzyme General Division common stock are
subject to all of the risks and uncertainties described in the those reports. We
caution investors not to place undue reliance on the forward-looking statements
contained in this press release. These statements speak only as of the date of
this press release, and we undertake no obligation to update or revise the
statements, except as may be required by law.
Genzyme(R) is a registered trademark of Genzyme Corporation. AldurazymeTM is a
trademark of BioMarin/Genzyme LLC.
# # #
Genzyme's press releases and other company information are available at
www.genzyme.com and by calling Genzyme's investor information line at
1-800-905-4369 within the United States or 1-703-797-1866 outside the United
States.
BioMarin's press releases and other company information are available at
www.biomarinpharm.com.