The compound, PTC124, has been granted orphan drug status by the Committee for Orphan Medicinal Products, a unit of the European Medicines Agency (EMEA). Such status is granted for products designed to provide relief from diseases affecting less than five in 10,000 people in Europe.
Orphan drug designation provides opportunities for free protocol assistance, fee reductions for access to the centralized community procedures before and after marketing authorization, and 10 years of market exclusivity following drug approval.
PTC124 is a novel, orally administered drug that targets nonsense mutations and is being investigated initially as a treatment for cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD), with the potential to treat a number of other genetic disorders. It is estimated that 10% of CF patients and 15% of DMD patients have these diseases as a consequence of nonsense mutations.
Results from phase I studies have confirmed that PTC124 is orally bioavailable, generally well tolerated, and achieves target plasma concentrations that have been associated with activity in preclinical models.
Pending regulatory approval PTC expects to advance PTC124 into phase II studies in patients with nonsense-mutation-mediated CF and DMD during the third quarter of 2005 in the US.