BioMarin Pharmaceutical’s Naglazyme has become the first approved treatment in the European Union for the rare genetic disease mucopolysaccharidosis VI.
Naglazyme (galsulfase) was first approved in the USA last year, and with European approval in place BioMarin is expected total product sales to be in the $28 million to $32 million range in 2006.
“With European commercial operations in place, we are ready to launch,” said the firm’s chief executive, Jean-Jacques Bienaime, referring to the company’s recently-opened European headquarters in London, UK. He also said that sales of BioMarin’s other commercialised product - Aldurazyme (laronidase) for the related condition MPS I – would be in the $90 million to $100 million range this year.
MPS VI is an enzyme deficiency that leads to progressive cellular, tissue and organ system dysfunction. In clinical trials, Naglazyme was shown to provide clinically important benefits for MPS VI patients, specifically, improved endurance as demonstrated by a 12-minute walk test and three-minute stair climb. The disease is rare, affecting an estimated 1,100 individuals in the developed world.