BioMarin’s GALNS – enzyme replacement therapy for Morquio Syndrome A

Posted by lifetech on March 15th, 2011

BioMarin has built a portfolio of products for rare orphan conditions, which have proven to be valuable commercial opportunities despite targeting diseases with low prevalence. BioMarin’s latest product, GALNS, is an enzyme replacement therapy (ERT) currently in phase III testing for MPS IVA, a rare genetic disease also called Morquio syndrome A. MPS IVA causes severe disability and early mortality, and current treatment options are of limited benefit. However, similar diseases have been successfully treated with ERT, including two products developed by BioMarin itself: Aldurazyme for MPS I and Naglazyme for MPS VI. Thus, there is good rationale for developing ERT in MPS IVA.

In a pilot phase I/II study, GALNS showed evidence of clinical activity, as measured by biomarkers, respiratory function and physical endurance. But the trial was small and uncontrolled, so these early results must be confirmed in the ongoing placebo-controlled phase III trial. If phase III proceeds as planned, we anticipate top-line results in the third quarter of 2012. GALNS would make a fine addition to BioMarin’s ERT portfolio. Of course, regulatory success hinges on phase III outcomes, and the potential for commercial success will also depend heavily on GALNS’ clinical performance in a controlled trial. In our recent report, we explore these issues in greater depth.