Please login to the form below

Not currently logged in

Pompe drug wins orphan status

The US Food and Drug Administration has granted orphan drug status to BioMarin Pharmaceutical's investigational therapy for Pompe disease

The US Food and Drug Administration (FDA) has granted orphan drug status to BioMarin Pharmaceutical's investigational therapy for Pompe disease, a rare lysosomal storage disorder that damages the heart and other muscles and is often fatal.

The drug, BMN-701, is a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) that is expected to begin phase I/II clinical studies in the first quarter of 2011.

Orphan drug status confers a number of benefits, including tax breaks, extra advice and assistance from the FDA, and seven years of marketing exclusivity if the product is approved. It was designed to encourage the development of treatments for diseases that affect fewer than 200,000 patients in the US.

BioMarin noted that the FDA had determined that its Pompe drug was sufficiently different from Genzyme's alglusidase alfa (Myozyme/Lumizyme) to allow BMN-701 to have its own orphan designation. Had the federal agency decided that the two treatments were not sufficiently different, BioMarin would have had to prove clinical superiority over alglusidase alfa in order to win the seven years of marketing exclusivity granted through the programme.

BioMarin added the experimental Pompe treatment to its pipeline when it acquired the privately-held biotech company ZyStor in mid-August. In vitro studies suggest that the product, which uses ZyStor's proprietary Glycosylation Independent Lysosomal Targeting (GILT) technology, could deliver markedly higher levels of enzyme to the lysosomes of muscle cells in Pompe patients than Genzyme's product does, according to BioMarin.

1st September 2010


COVID-19 Updates and Daily News

Featured jobs


Add my company
CSafe Global

CSafe Global is the only global provider of a full line of cold chain solutions and is the world’s largest...

Latest intelligence

Has the pandemic opened up a future of accelerated diagnosis and better care for rare disease patients?
The challenge with rare disease is in the name – it's rare, so awareness is limited and diagnosis hindered. Could a more virtual existence change this? A Medical Affairs viewpoint...
The other side of … blood cancer
To stay motivated for the fight, some patients need to feel like active players in the treatment journey, not passengers to every decision....
COVID clinical trial
COVID-19 – a catalyst for technology adoption in clinical trials
As the COVID-19 crisis disrupts clinical trials around the globe, it is also proving to be a catalyst that may transform its approach to trials for good...