BioMarin Announces FDA Approval for VIMIZIM(TM) for the Treatment of Patients With Morquio A Syndrome

In Press Release by Cameron

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February 14, 2014

VIMIZIM is the First and Only Specific Treatment for Patients With This Ultra-Rare Genetic Condition

BioMarin Pharmaceutical Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved VIMIZIM™ (elosulfase alfa) for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

“The FDA approval of VIMIZIM is an important milestone for BioMarin and for patients with Morquio A syndrome. VIMIZIM is the first and only therapy designed to address the condition at the cellular level, fulfilling a large unmet medical need for patients and their families,” said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. “With the approval of VIMIZIM, BioMarin firmly establishes its leadership in advancing therapies to treat MPS diseases. We have developed three therapies to treat three different MPS diseases and continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases.”

VIMIZIM is an enzyme replacement treatment for Morquio A syndrome, which affects an estimated 3,000 patients in the developed world. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism. The pervasive and progressive accumulation of GAGs leads to significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality of life, and early mortality. The most common features of the disease are progressive skeletal dysplasia, the need for frequent surgical procedures related primarily to musculoskeletal or respiratory dysfunction, and significant limitations in mobility, endurance, and breathing.

“In clinical trials, VIMIZIM was shown to significantly improve endurance, which possibly could change the course of the disease. As a treating physician, I am encouraged that the therapy has proven to provide clinical benefit, which is not always possible to demonstrate with ultra-rare diseases,” said Paul Harmatz, M.D., Associate in Gastroenterology and Nutrition at the Children’s Hospital and Research Center in Oakland, California and clinical investigator in the VIMIZIM Phase 3 trial. “The approval of VIMIZIM is an important advance for Morquio A patients and their families and moves treatment beyond supportive care to treating the underlying cause of the disease.”

“We are thrilled that patients with Morquio A syndrome will have access to this potentially life-changing therapy and appreciate BioMarin’s commitment to the MPS community and the individuals and their families who are affected by these devastating conditions,” said Barbara Wedehase, MSW, CGC, Executive Director of the National MPS Society. “Until now, patients with Morquio A syndrome didn’t have a drug treatment option. This approval provides the community with a therapy and with hope.”

Shipments of VIMIZIM to the distribution channels will commence immediately, and BioMarin will begin promotion of VIMIZIM in the U.S. immediately. BioMarin has also submitted marketing applications for VIMIZIM in the European Union, Brazil, Australia,  Canada, and Mexico.

Find the full press release here.