BioMarin Pharmaceutical Inc. recently announced its program for its third enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis IVA (MPS IVA), or Morquio A Syndrome. BioMarin plans to initiate a Phase 1/2 clinical trial in the first quarter of 2009. “With two MPS drugs on the market, we plan to leverage our clinical, manufacturing and regulatory expertise to efficiently develop a treatment for Morquio patients,” said Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin. “Our planned program includes a clinical assessment study to measure the depth and breadth of disease as well as a separate GALNS Phase 1/2 study in Morquio Type A patients. Preliminary studies are promising and indicate that our drug candidate binds naturally to bone matrix and can adequately reach the growth cartilage after IV infusion. The skeletal system disease is a primary concern in the treatment of this disease.” The company has successfully developed and manufactures two FDA-approved enzyme replacement therapies for the treatment of MPS I and MPS VI. Naglazyme(R) (galsulfase) MPS VI is wholly developed and commercialized by BioMarin. Aldurazyme(R) (laronidase) for MPS I is manufactured by BioMarin and marketed by Genzyme Corporation.
