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Last updated on April 17, 2014 at 1:21 EDT

BioMarin Initiates Clinical Assessment Program for Morquio A Syndrome

November 4, 2008

BioMarin Pharmaceutical, a developer and marketer of biopharmaceuticals, has initiated the Morquio clinical assessment program for patients with the lysosomal storage disease Mucopolysaccharidosis type IVA or Morquio A syndrome.

The Morquio clinical assessment program (MorCAP) is designed to augment available data on the disease by measuring endurance and respiratory function and other parameters in affected patients. BioMarin expects to follow the MorCAP program with a Phase Ib clinical trial of enzyme replacement therapy beginning in the first quarter of 2009.

The primary objectives of the Phase Ib study will be to evaluate safety and to establish the optimal dose of enzyme based on pharmacokinetic and pharmacodynamic parameters.

Emil Kakkis, chief medical officer of BioMarin, said: “After successfully advancing two enzyme replacement therapies in approximately five years each from investigational new drug filing to FDA approval, we plan to leverage our clinical, manufacturing and regulatory expertise to develop a treatment for Morquio A syndrome.

“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. We recently have also shown that GALNS can reduce keratan sulfate storage in Morquio chondrocytes. This is important as the skeletal system is a primary concern in the treatment of this disease.”