BioMarin Announces Health Canada Approval of VIMIZIM™ (elosulfase alfa) for the Treatment of Morquio A Syndrome

Approval delivers first pharmaceutical therapy for children and adults with ultra-rare, life-limiting disorder

TORONTO, July 7, 2014 /CNW/ - BioMarin Pharmaceutical Inc. BMRN announced today that Health Canada has approved VIMIZIM™(elosulfase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome. The approval makes VIMIZIM the first and only pharmaceutical treatment option available in Canada for children and adults living with this severely debilitating, progressive and life-limiting disorder.

"Morquio A is an ultra-rare disease for which there is no cure. In fact, until now, available treatments only addressed the symptoms of the disease," said Dr. John Mitchell, a leading pediatric endocrinologist and biochemical geneticist based in Montreal, and clinical investigator in the VIMIZIM Phase 3 trial. "The approval of VIMIZIM fills a critical unmet need for Morquio A patients and their families as it moves treatment beyond supportive care to addressing the underlying cause of the disease."

The New Drug Submission for VIMIZIM was submitted to Health Canada in October 2013 under Priority Review Status, which allows for the "fast-tracking" of submissions intended for the treatment, prevention or diagnosis of serious, life-threatening or severely debilitating diseases or conditions.

"The Health Canada approval of VIMIZIM is a significant milestone for BioMarin, and for Canadians living with Morquio A and their families," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "To date, BioMarin has developed treatments for three different MPS diseases. The approval of VIMIZIM firmly establishes our leadership in advancing important therapies for the treatment of MPS diseases. We will continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases."

About VIMIZIM
VIMIZIM (elosulfase alfa) is an enzyme replacement therapy (ERT) for the treatment of patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA). VIMIZIM is the first and only ERT designed to target the underlying cause of Morquio A - a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Infused ERT with VIMIZIM replaces deficient GALNS activity to minimize progressive multi-systemic manifestations.¹

VIMIZIM is approved for the treatment of patients with Morquio A in Canada, the United States, and the European Union.

About Morquio A
Morquio A is a disease in which people have deficient activity of an enzyme that is essential in the breakdown and removal of the mucopolysaccharides called keratan sulfate.^1,2 The incompletely broken down mucopolysaccharides remain stored in cells in the body causing progressive damage.² 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.^3,4,5 The disease substantially limits both the quality and length of life of those affected.⁶

The rate of incidence of Morquio A is as yet unconfirmed and varies among different populations, and estimates vary between 1 in 76,000 live births and 1 in 640,000 live births.⁶ The incidence rate in Canada is estimated at 0.33-0.5 per 100,000 live births.⁷ Globally, the estimated prevalence is approximately 3,000 patients in the developed world.⁸

About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises five 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) Powder for Oral Solution and Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS); and VIMIZIM™ (N-acetylgalactosamine 6-sulfatase) for the treatment of Morquio A (MPS IVA). Product candidates include: BMN 165 (PEGylated recombinant phenylalanine ammonia lyase), also referred to as PEG-PAL, 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 3 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 www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

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.

References
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¹ The Canadian Society for Mucoploysaccaride & Related Diseases Inc. A Guide to Understanding Mucopolysaccharidosis (MPS) IV.
² Mayo Medical Laboratories. Mucopolysaccharides (MPS) Screen, Urine. Available at: www.mayomedicallaboratories.com/test-catalog/Clinical+and+Interpretive/84464. Accessed on February 26, 2014.
³ Borlot et al. Mucopolysaccharidosis type IVA: Evidence of primary and secondary central nervous system involvement. American Journal of Medical Genetics Part A. Available at: http://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.36424/abstract. Access on February 26, 2014.
⁴ Regier, Debra S et al. Mucopolysaccharidosis Type IVA. Available at: www.ncbi.nlm.nih.gov/books/NBK148668/. Accessed on February 26, 2014.
⁵ Medline Plus. U.S. National Library of Medicine - National Institutes of Health. Morquio syndrome. Available at: www.nlm.nih.gov/medlineplus/ency/article/001206.htm. Accessed on February 26, 2014.
⁶ Nelson J. Incidence of the mucopolysaccharidoses in Northern Ireland. Hum Genet. 1997; 101:355-358.
⁷ National MPS Society. A guide to understanding MPS IV. Published 2008. Available at: http://www.mpssociety.org/wp-content/uploads/2011/07/booklet_MPS_IV_v6.pdf. Accessed September 2012.
⁸ Regroupement québécois des maladies orphelines (RQMO). RQMO joins Morquio A Community to mark International MPS Awareness Day. Available at: http://www.newswire.ca/en/story/1356147/rqmo-joins-morquio-a-community-to-mark-international-mps-awareness-day. Accessed on: May 27, 2014.

SOURCE BioMarin