BioMarin Pharmaceutical Inc., develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions, has completed the phase III enrollment for N-acetylgalactosamine 6-sulfatase (GALNS or BMN-110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. The company expects to report results in the fourth quarter of 2012.

“It is a major milestone in the GALNS programme to have 176 patients randomized and dosed at 31 sites in 17 countries worldwide. We exceeded the planned sample size of 162 patients because physician and patient demand for participation in the trial was extraordinarily high. We were able to accommodate this excess without any delay in our plans to have data available in the fourth quarter this year,” said Hank Fuchs, MD, chief medical officer of BioMarin. “We would like to thank the investigators, patients and their families, and hardworking employees of BioMarin who have contributed efforts to the largest study in the history of the company. The phase III trial for GALNS is currently the highest development priority at BioMarin, and we have taken every possible measure to design and conduct this study under the highest standards to maximize our chance of success. Along with our supplemental studies for patients under five years of age and non-ambulatory patients, we believe the results from the phase III pivotal study will be well-positioned to support regulatory filings in the first quarter of 2013.”

The phase III trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of GALNS in patients with MPS IVA. The study is exploring doses of two mg/kg/week and two mg/kg/every other week for a treatment period of 24 weeks. The primary endpoint is the six-minute walk test, and the secondary endpoints are the three-minute stair climb test and urine keratan sulfate concentration.