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BioMarin up on Vimizim briefing docs

November 16, 2013 2:25 AM UTC

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) rose $4.96 to $69.29 on Friday after FDA reviewers said the company's Vimizim elosulfase alfa was effective to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome) and raised no major safety concerns. Reviewers said Vimizim led to a "modest improvement" on the six-minute walk test (6MWT), the primary endpoint, in the Phase IIII MOR-004 trial, though the reviewers said based on the data Vimizim may be more effective in patients with more severe disease. The reviewers said adverse reactions with Vimizim, including anaphylaxis, were similar to those with other enzyme replacement therapies. The reviewers did note that the majority of patients in MOR-004 developed neutralizing antibodies against Vimizim and said "further studies may be needed to better understand the role of antibody development on long-term safety and efficacy." The comments came in briefing documents released ahead of Tuesday's FDA's Endocrinologic and Metabolic Drugs Advisory Committee meeting to discuss Vimizim. The recombinant human N-acetylgalactosamine-6-sulfatase has a Feb. 28 PDUFA date. ...