GALNS (BioMarin) success in Phase III study for MPS IVA

BioMarin Pharmaceutical Inc.has announced that the pivotal Phase III study of GALNS met the primary endpoint of change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS at the dose of 2 mg/kg (p=0.0174). MOR-004 was a randomized, double-blind, placebo-controlled study evaluating two doses of GALNS (BMN-110, N- acetylgalactosamine-6-sulfatase) for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.

Patients dosed with GALNS at 2 mg/kg every other week did not show a meaningful or statistically significant change from baseline compared to placebo. The company also announced preliminary data from the MOR-005 extension study which suggests that clinical benefits continue to improve with further dosing with GALNS. Only a limited number of patients have reached the 36 or 48 week points of total time on treatment in the extension study, and the results will be updated when the study is completed.

The company confirmed that based on the results of MOR-004, and following planned discussions with regulatory authorities, it expects to submit marketing applications starting in the first quarter of 2013.