BioMarin Pharmaceutical Inc. announced the initiation of the Morquio Clinical Assessment Program (MorCAP) for patients with the lysosomal storage disease Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. 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 programme with a phase 1b clinical trial of enzyme replacement therapy beginning in the first quarter of 2009. The primary objectives of the phase 1b study will be to evaluate safety and to establish the optimal dose of enzyme based on pharmacokinetic and pharmacodynamic parameters.
"After successfully advancing two enzyme replacement therapies in approximately five years each from IND filing to FDA approval, we plan to leverage our clinical, manufacturing and regulatory expertise to develop a treatment for Morquio A syndrome," said Emil Kakkis, M.D., Ph.D., chief medical officer of BioMarin. "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."
"We are excited to be the first site to enroll patients in the BioMarin Clinical Assessment Program for MPS IVA patients. This study is crucial to developing a deeper understanding of the clinical outcomes for this rare disorder, which will help lead to better disease management and therapy options, said Barbara Burton, M.D., director, MPS/ML Treatment Program, Children's Memorial Hospital."
BioMarin has developed and manufactures two FDA-approved enzyme replacement therapies, one for the treatment of MPS I and one for the treatment of MPS VI. Naglazyme (galsulfase) for MPS VI is wholly developed and commercialized by BioMarin. Aldurazyme (laronidase) for MPS I is manufactured by BioMarin and marketed by Genzyme Corporation.
Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disease characterized by deficient activity of N-acetylgalactosamine-6- sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS). This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the thorax as well as macrophage storage in the lung likely impairs respiratory function and contributes to sinopulmonary infections. Odontoid hypoplasia and ligamentous laxity can commonly cause cervical spinal instability and potentially spinal cord compression. Other symptoms may include recurrent infections, hearing loss, corneal clouding, and heart valvular disease. Initial symptoms often become evident in the first five years of life. Depending on severity of the disease, age of diagnosis will vary. Many patients end up wheelchair dependent in their second decade of life and undergo numerous surgeries to manage their disease.
The rate of incidence of MPS IVA is as yet unconfirmed, but estimates vary between 1 in 200,000 live births to 1 in 300,000 live births. Approximately 400 patients worldwide are currently registered in a public registry, based on their publications. The prevalence of patients with MPS IVA appears substantially higher than that with MPS VI based on published reports.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and preclinical product candidates.