BioMarin Submits VIMIZIM™ (elosulfase alfa) NDS to Health Canada for the Treatment of Morquio A Syndrome Under Priority Review Status

Approval would deliver first treatment for Canadians living with ultra-rare, genetic metabolic disorder

TORONTO, Dec. 4, 2013 /CNW/ - BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it submitted a New Drug Submission (NDS) for VIMIZIM™ (elosulfase alfa) under Priority Review status. VIMIZIM is an investigational enzyme replacement therapy for the treatment of patients with the lysosomal storage disorder Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA). Approval of VIMIZIM would make it the first available drug treatment option for Canadians living with this rare genetic metabolic disorder.

"Morquio A is an extremely rare disease that substantially limits both the quality and length of life for those affected by it. There is no cure, and current available treatments are primarily symptomatic," said Dr. Julian Raiman, a leading pediatric metabolic geneticist based in Toronto. "The potential approval of VIMIZIM would fill a critical unmet need for patients living with this severely debilitating, life-limiting and progressive disease."

Health Canada designates Priority Review to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. Priority is intended to accelerate the regulatory review.

"Every day, Canadians living with Morquio A face seemingly insurmountable challenges. We are encouraged that children who are born with this disorder could soon have a drug therapy option that treats the root cause of the disease," said Kirsten Harkins, Executive Director, The Canadian Society for Mucopolysaccharide and Related Diseases Inc. (The Canadian MPS Society).

"BioMarin has submitted the NDS to Health Canada based on the results from our Phase 3 pivotal study, which we believe demonstrates that VIMIZIM offers a substantial benefit to patients with Morquio A," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "With this application, BioMarin continues in its long-standing tradition of advancing important therapies for those who are most in need."

VIMIZIM (elosufase alfa) is an investigational treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA), which is currently under review by the FDA. VIMIZIM is the first and only enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome - a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Infused enzyme replacement therapy with VIMIZIM replaces deficient GALNS activity to minimize progressive multi-systemic manifestations. Morquio A syndrome is a rare, severely debilitating and progressive disease that currently has no standard accepted treatment other than supportive care.

About Morquio A Syndrome
Morquio A Syndrome, or mucopolysaccharidosis IVA (MPS IVA), is a disease in which people are missing an enzyme that is essential in the breakdown and removal of the mucopolysaccharides called keratan sulfate. The incompletely broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.

The rate of incidence of Morquio A syndrome is as yet unconfirmed and varies among different populations, and estimates vary between 1 in 200,000 live births and 1 in 450,000 live births. The estimated prevalence is approximately 3,000 patients in the developed world. 

About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include VIMIZIM (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase 3 clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 3 clinical development for the treatment of PKU, BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, BMN 701, a novel fusion of acid alpha glucosidase (GAA) with a peptide derived from insulin like growth factor 2, which is currently in Phase 1/2 clinical development for the treatment of Pompe disease, BMN 111, a modified C-natriuretic peptide, which is currently in Phase 1 clinical development for the treatment of achondroplasia, and BMN 190, a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease. For additional information, please visit 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: expectations regarding the NDS filing for Vimizim with Health Canada; the potential outcome of the review of such filings; and the possible approval of such product candidates. 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: results and timing of current and planned clinical trials of its product candidates; the nature of any questions or responses from Health Canada; compliance by Health Canada with the priority review guidelines; the content and timing of decisions by regulatory agencies, including Health Canada; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2012 Annual Report on Form 10-K, as amended, and the factors contained in BioMarin's reports on Form 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.

VIMIZIM™ is our trademark, and BioMarin®, Naglazyme®, Kuvan®, Firdapse® are registered trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

SOURCE: BioMarin

For further information:

Beth Daniher
Cohn & Wolfe
416-924-5700 ext. 4070

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