BioMarin gets Europeon approval for rare-disease treatment

SAN RAFAEL -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) on Monday said the European Commission has granted marketing authorization for its treatment of Morquia synrome, a rare inherited metabolic disease.

The commission approved Vimzim to treat patients of all ages. BioMarin described the drug as “the first drug ever approved” for the syndrome, in which the body is lacking in a substance needed to break down chains of sugar molecules. The approval grants Vimzim orphan status in the European Union, meaning BioMarin will have 10 years of market exclusivity, the company said.

"This approval of Vimzim in Europe is a key milestone, because we estimate that 85 percent of Morquio A or MPS IVA patients live outside of the United States," said Jean-Jacques Bienaime, chief executive officer. “We continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases.”

The U.S. Food and Drug Administration approved Vimzim for patients with Mucopolysaccharidosis type IVA (MPS IVA, also known as Morquio A syndrome) on Feb. 14. BioMarin has also submitted marketing applications for Vimzim in Brazil, Australia, Canada, Mexico and Japan.

The drug is an enzyme-replacement therapy for patients with Morquio A syndrome. It's an ultrarare, severely debilitating lysosomal storage disease that affects an estimated 3,000 patients in the developed world, according to BioMarin.

A deficiently active enzyme involved in glycosaminoglycan metabolism can lead to “significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality of life, and early mortality,” BioMarin said.

The most common features of the disease are progressive skeletal dysplasia (dwarfism), the need for frequent surgical procedures related primarily to musculoskeletal or respiratory dysfunction, and significant limitations in mobility, endurance and breathing.

The drug recently underwent clinical trials, in which safety and efficacy were assessed in a 24-week, randomized, double-blind placebo-controlled trial on 176 patients ages 5--57 with the disorder. Patients who received weekly infusions of the drug showed significant improvement in their ability to walk, according to BioMarin.  

"Vimzim addresses the condition at the cellular level, fulfilling a large unmet medical need and represents an advance for Morquio A patients and their families," said Christian Hendriksz of Salford Royal NHS Foundation Trust and lead investigator for the phase 3 clinical trial. "As a treating physician, I am encouraged that the therapy shifts the treatment beyond supportive care to treating the underlying cause of the disease, potentially changing the course of this devastating disease."

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