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BioMarin Announces FDA Approval for VIMIZIM(TM) (elosulfase alfa)

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


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BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) 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.

BioMarin will offer support to patients through its BioMarin Patient & Physician Support (BPPS) team. Through BPPS, patients receive live, personalized support by a specialized case manager who will research insurance coverage and alternative benefit options. BPPS will help patients obtain coverage and minimize out-of-pocket expenses and find alternative financial assistance for treatment. To reach a BPPS case manager, please call, toll-free, 1-866-906-6100 or e-mail bpps@bmrn.com. For more information about VIMIZIM, please visit www.VIMIZIM.com.

Clinical Trial Results

The safety and efficacy of VIMIZIM were assessed in a 24-week, randomized, double-blind, placebo-controlled clinical trial of 176 patients with MPS IVA (MOR-004). The primary endpoint of the trial, change in six-minute walk distance at 24 weeks, was statistically significant in patients receiving weekly infusions of VIMIZIM at the dose of 2 mg/kg with a mean increase of 22.5 meters (p=0.0174) over placebo.

In patients who continued to receive VIMIZIM 2 mg/kg once per week for another 48 weeks (for a total of 72-week exposure), walking ability was sustained to a similar level that was achieved during the first 24 weeks of treatment in the placebo-controlled trial, MOR-004.

Overall, sustained improvements across multiple efficacy measurements and across multiple clinical trials provided evidence of clinical benefit to patients with MPS IVA, a chronic, progressive disease in which clinical deterioration is the expected course.

The adverse events observed in clinical trials were similar to those seen in other enzyme replacement therapies. In the Phase 3 trial, the most common adverse reactions (=10% and a higher incidence than placebo) that occurred were pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue. No new types of adverse reactions were reported in the Phase 3 extension trial. The most common adverse reactions (=10%) observed across pre-marketing clinical trials were similar in type and frequency as those observed in the placebo-controlled trial. Acute reactions requiring intervention were managed by either temporarily interrupting or discontinuing infusion, and administering additional antihistamine, antipyretics, or corticosteroids.

Note to Investors

BioMarin will host a webcast to discuss the VIMIZIM approval Tuesday, February 18, 2014 at 5:00 a.m. PT. Dial-in information for the conference call will be distributed prior to the call.

Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.BMRN.com. A replay of the call will be archived on the site for one week following the call.


VIMIZIM (elosulfase alfa) is a treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA). VIMIZIM is the first enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome – a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). VIMIZIM is intended to provide the exogenous enzyme GALNS that will be taken up into the lysosomes and increase the catabolism of GAGs. Morquio A syndrome is a rare, severely debilitating and progressive disease that previously had no standard accepted treatment other than supportive care.

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