Final Approval Anticipated in Early 2006
NOVATO, Calif., Sept. 15 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical
Inc. (Nasdaq and SWX: BMRN) announced today that the Committee for Medicinal
Products for Human Use (CHMP), the scientific committee of the European
Medicines Evaluation Agency (EMEA), has issued a positive opinion on the
company's Marketing Authorization Application for Naglazyme(TM) (galsulfase)
an investigational enzyme replacement therapy for treatment of
mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome).
The committee's proposed label states that Naglazyme is approved in the
European Union as a long-term enzyme replacement therapy in patients with a
confirmed diagnosis of MPS VI to treat the clinical manifestations of the
disease. All clinical post-authorization commitments requested by the CHMP
will be fulfilled through a clinical surveillance program that will monitor
patients on commercial therapy. No additional clinical trials are required.
"We are pleased that the CHMP has recommended approval of Naglazyme for
MPS VI and look forward to receiving a final determination from the European
Commission in the next three to four months," stated Jean-Jacques Bienaime,
Chief Executive Officer of BioMarin. "Our effort to establish a small,
Europe-based sales force to market Naglazyme is well under way and, pending
final approval from the European Commission, we expect to initiate product
launch in early 2006."
The positive opinion from the CHMP is the final step before formal
approval to market Naglazyme in the 25 member states of the European Union,
Iceland and Norway. The CHMP has advised BioMarin that the committee's
opinion will be forwarded to the European Commission (EC), which is expected
to make a formal decision on marketing authorization within three to four
months. The EC generally follows the advice of the CHMP, but it is not
obligated to do so.
The CHMP is a scientific body made up of representatives from the 25
member states of the European Union, Iceland and Norway. The CHMP reviews
medicinal product applications on their scientific and clinical merit, and
provides advice on their approval to the EC, including recommendations about
product labeling.
The CHMP reviewed BioMarin's clinical data for Naglazyme, which the
company submitted in December 2004. The data included were from a 24-week,
Phase 3, multi-center, double-blind, placebo-controlled trial involving 39
patients, of which one patient from the placebo group dropped out of the trial
for reasons unrelated to treatment, and 24 weeks of data from the extension
study, which involved all remaining 38 patients.
About MPS VI
MPS VI (also known as Maroteaux-Lamy syndrome) is a debilitating,
life-threatening genetic disease caused by a deficiency of the enzyme
N-acetylgalactosamine 4-sulfatase. This enzyme deficiency leads to the
accumulation of certain complex carbohydrates, glycosaminoglycans (GAGs), in
the lysosomes, giving rise to progressive cellular, tissue and organ system
dysfunction. The majority of individuals with MPS VI die from disease-related
complications between childhood and early adulthood. Additional information
can be found at http://www.mpsvi.com.
About Naglazyme
Naglazyme is an enzyme replacement therapy for the treatment of MPS VI.
On May 31, 2005, the U.S. Food and Drug Administration (FDA) granted market
authorization of Naglazyme for patients with MPS VI. As the first drug
approved for this indication, the FDA granted Naglazyme orphan drug status,
which confers upon it seven years of market exclusivity. In Europe,
regulatory authorities have designated Naglazyme as an orphan medicinal
product, which, if it is the first therapy approved for MPS VI, will guarantee
10 years of market exclusivity within the European Union. Additional
information can be found at http://www.naglazyme.com.
Naglazyme is indicated for patients with MPS VI. Naglazyme has been shown
to improve walking and stair-climbing capacity.
The most common adverse events observed in clinical trials in Naglazyme-
treated patients were headache, fever, arthralgia, vomiting, upper respiratory
infections, abdominal pain, diarrhea, ear pain, cough, and otitis media.
Severe reactions included angioneurotic edema, hypotension, dyspnea,
bronchospasm, respiratory distress, apnea, and urticaria. The most common
symptoms of infusion reactions included fever, chills/rigors, headache, rash,
and mild to moderate urticaria. Nausea, vomiting, elevated blood pressure,
retrosternal pain, abdominal pain, malaise, and joint pain were also reported.
No patients discontinued Naglazyme infusions for adverse events and all
patients that completed the double-blind portion of the trial continue to
receive weekly infusions of Naglazyme. Nearly all patients developed
antibodies as a result of treatment, but the level of the immune response did
not correlate with the severity of adverse events or impact the improvements
experienced in endurance. Because antihistamine use may increase the risk of
apneic episodes, evaluation of airway patency should be considered prior to
the initiation of treatment. Consideration to delay Naglazyme infusion should
be given when treating patients who present with an acute febrile or
respiratory illness.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for
serious diseases and medical conditions. The company's product portfolio is
comprised of three approved products and multiple clinical and preclinical
product candidates. Approved products include Naglazyme(TM) (galsulfase) for
mucopolysaccharidosis VI (MPS VI), a product wholly developed and
commercialized by BioMarin, Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), and Orapred(R) (prednisolone sodium phosphate
oral solution) for inflammatory conditions. Investigational product
candidates include Phenoptin(TM) (sapropterin hydrochloride), a Phase 3
product candidate for the treatment of phenylketonuria (PKU). For additional
information, please visit http://www.BMRN.com. Information on BioMarin's website is
not incorporated by reference into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: the development and commercialization of Naglazyme and
Phenoptin; expectations related to post-marketing commitments for Naglazyme;
and actions by regulatory authorities. These forward-looking statements are
predictions and involve risks and uncertainties such that actual results may
differ materially from these statements. These risks and uncertainties
include, among others: possible delays in launching Naglazyme in the E.U. and
slow market penetration in the U.S. and E.U.; the content and timing of
decisions by the FDA and European Commission and other regulatory authorities
concerning Naglazyme and Phenoptin; issues or complications associated with
post-marketing commitments; the results of current and future clinical trials
of Phenoptin; and those factors detailed in BioMarin's filings with the
Securities and Exchange Commission, including, without limitation, the factors
contained under the caption "Factors That May Affect Future Results" in
BioMarin's 2004 Annual Report on Form 10-K and the factors contained in
BioMarin's reports on Forms 10-Q and 8-K. Stockholders are urged not to place
undue reliance on forward-looking statements, which speak only as of the date
hereof. BioMarin is under no obligation, and expressly disclaims any
obligation, to update or alter any forward-looking statement, whether as a
result of new information, future events or otherwise.
NOTE: Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.
Orapred(R) is a registered trademark of Medicis Pediatrics, Inc. and is
used under license.
Contacts:
Investors Media
Joshua A. Grass Susan Ferris
Director, Business Development Manager, Corporate
& Finance Communications
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
415-506-6777 415-506-6701
SOURCE BioMarin Pharmaceutical Inc.
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Related links:
http://www.bmrn.com
CONTACT:
investors, Joshua A. Grass, Director,
Business Development & Finance, +1-415-506-6777, or media, Susan
Ferris, Manager, Corporate Communications, +1-415-506-6701, both
of BioMarin Pharmaceutical Inc.
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