BioMarin Pharmaceutical was celebrating yesterday after the US regulator approved the firm’s Naglazyme (galsulfase) as the first specific therapy for the treatment of MPS VI (also known as mucopolysaccharidosis VI and Maroteaux-Lamy syndrome) – a debilitating and life-threatening genetic disease.

As the first drug ever approved for this condition, which affects an estimated 1,100 individuals in the developed world, Naglazyme has been granted orphan drug status in the US, giving BioMarin up to seven years of market exclusivity. The firm is planning to launch the drug in around 30 days.

MPS VI is an enzyme deficiency that leads to progressive cellular, tissue and organ system dysfunction. In clinical trials, Naglazyme was shown to provide clinically important benefits for MPS VI patients, specifically, improved endurance as demonstrated by a 12-minute walk test and three-minute stair climb.

An application to market Naglazyme is currently pending in the European Union. A decision on approvability in this market is expected in the fourth quarter of the year, with final approval possible in early 2006.