SAN RAFAEL, Calif., Nov. 15, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the French National Agency for Medicines and Health Products Safety (ANSM) has granted an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use, for patient sales of Vimizim for the treatment of Morquio A Syndrome. An ATU is the regulatory mechanism used by the ANSM to make non-approved drugs available to patients in France when a genuine public health need exists. This ATU allows patients with Morquio A Syndrome in France to receive treatment with Vimizim before marketing authorization for the product is granted in the European Union. Government allocations to hospitals allow payment for Vimizim for patients included in the ATU program. The company expects to book revenue on named-patient basis sales and will continue to assist patients in France wherever possible prior to full market product approval in the European Union.
"With a significant number of all Morquio A patients living throughout Europe, we are pleased to be able to provide Vimizim to patients in France under the ATU program," said Jeff Ajer, Senior Vice President, Global Commercial Operations at BioMarin. "We look forward to working closely with European regulatory authorities through the final stages of full market approval in this region."
About Morquio A SyndromeMucopolysaccharidosis IVA (MPS IVA, also known as Morquio A syndrome) is a disease characterized by deficient activity of Nacetylgalactosamine- 6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.
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