BioMarin provides pipeline update

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said during its R&D day on Tuesday that it expects FDA to convene an advisory committee on Nov. 19 to discuss a BLA for Vimizim elosulfase alfa to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome). Vimizim, a recombinant human N-acetylgalactosamine-6-sulfatase, has a Feb. 28, 2014, PDUFA date. FDA granted Priority Review to the BLA, but the agency subsequently extended the PDUFA date by three months after BioMarin provided additional CMC information the agency had requested during the initial review of the BLA. An MAA is under review in the EU.

BioMarin said it plans to start in 4Q13 or 1Q14 a Phase III trial of BMN-701 to treat late-onset Pompe's disease in patients who have previously been treated with alglucosidase alfa. The biotech said FDA has agreed on maximal inspiratory pressure (MIP) as a primary endpoint, but discussions regarding the primary endpoint and study design with European regulators are ongoing. BMN-701 is a fusion of insulin-like growth factor-2 (IGF-2) and acid alpha glucosidase (GAA) that uses Glycosylation Independent Lysosomal Targeting (GILT) technology. ...