Biomarin gets EU approval for genetic disease drug Naglazyme
The European Commission has granted marketing authorisation to California-based BioMarin Pharmaceutical for Naglazyme (galsulfase), the first treatment approved in the European Union (EU) for the genetic disease mucopoly saccharidosis VI (MPS VI or Maroteaux-Lamy syndrome).
The European Commission has granted marketing authorisation to California-based BioMarin Pharmaceutical for Naglazyme (galsulfase), the first treatment approved in the European Union (EU) for the genetic disease mucopoly saccharidosis VI (MPS VI or Maroteaux-Lamy syndrome).
As the first drug ever approved for MPS VI, Naglazyme has been granted orphan drug status in the EU, providing 10 years of market exclusivity.
MPS VI is a debilitating, life-threatening genetic disease caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase. This deficiency leads to progressive cellular, tissue and organ system dysfunction and death from disease-related complications between childhood and early adulthood.
Developer BioMarin will launch Naglazyme in the EU on a country-by-country basis. Jean-Jacques Bienaime, ceo of BioMarin said: 'With European commercial operations in place, we are ready to launch Naglazyme. Additionally, we are now positioned to partner with companies looking to bring other products for rare diseases to the European marketplace.'
BioMarin estimates sales of Naglazyme for 2006 to be in a range of $28m - $32m.
Ed Wraith, consultant pediatrician at the Royal Manchester Children's Hospital located in Manchester, UK, and a principal investigator for Naglazyme clinical trials, said: 'I have observed the dramatic improvement enzyme replacement therapy with Naglazyme can have on patients afflicted with MPS VI and am pleased that the therapy will soon be available to individuals outside of clinical studies'.