BioMarin phase 3 results send shares soaring

Hannah Blake

pharmaphorum

BioMarin Pharmaceuticals has announced that the phase 3 clinical trials of the company’s experimental drug, GALNS, met its primary endpoint. GALNS is being trialed for the treatment of patients with the rare lysosomal storage disorder, Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. The primary endpoint was in improving patients’ walking ability, which was found to be statistically significant in a six-minute walk distance compared with placebo at 24 weeks in patients receiving weekly infusions of GALNS at the dose of 2 mg/kg.

However, patients who received the same dose of GALNS every other week did not show any meaningful or statistically significant change compared to the placebo.

"The positive results from this pivotal study will help support GALNS as the first therapy available to help the approximate 3,000 people worldwide suffering from MPS IVA -- a rare, degenerative, life-threatening genetic condition with no available therapy. We are very pleased with the clarity that the MOR-004 study has provided us with respect to the appropriate dosing of GALNS.

“ We look forward to reviewing the results of this study with regulatory authorities, and applying for marketing authorizations starting in the first quarter of 2013."

Hank Fuchs, M.D., Chief Medical Officer at BioMarin.

Based on these positive clinical trial results of GALNS, BioMarin plans to seek regulatory approval in the first quarter of 2013. Shares in BioMarin increased by 30% following the announcement of this data.

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