BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has completed review of the Clinical Trial Application (CTA) and issued a notice of acceptance for the Phase 3 trial of N-acetylgalactosamine 6-sulfatase (GALNS) for the treatment of Mucopolysaccharidosis IVA (MPS IVA). The company expects to initiate the Phase 3 pivotal trial early in the first quarter of 2011.
"This important milestone reinforces our confidence in the Phase 3 trial design for GALNS as we move another step forward in the development of a treatment option for MPS IVA," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Results from the successful Phase 1/2 trial, along with our ongoing dialogue with health authorities in Europe and the U.S., have helped inform the design of a robust Phase 3 trial to maximize the therapy's chance for success."
The proposed Phase 3 trial would be a randomized, double-blind study with three study arms: placebo, two mg/kg every week and two mg/kg every other week in approximately 160 patients worldwide. The primary endpoint would be 6-minute walk distance with an entry criteria of baseline walk distance no greater than 325 meters. The company has submitted a special protocol assessment (SPA) to the FDA, with a response expected by late January.
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