US orphan status for Mirati's mocetinostat and Alexion's Soliris

USA-based Mirati Therapeutics (Nasdaq: MRTX) has been granted Orphan Drug designation by the US Food and Drug Administration for mocetinostat as a treatment for myelodysplastic syndrome (MDS).

Mocetinostat is being developed in Phase II clinical studies in combination with Vidaza (azacitidine, from Celgene) as a treatment for intermediate and high-risk MDS, as well as a single agent treatment in patients with diffuse large B-cell lymphoma (DLBCL) and bladder cancer targeting specific genetic mutations in histone acetylation that increase the likelihood of response in tumor cells.

"Orphan designation is an important piece of the development plan for mocetinostat as we evaluate combination and single agent clinical development opportunities for the program," said Charles Baum, president and chief executive of Mirati. "We are excited about the opportunity to identify and select patients whose cancers may be especially sensitive to mocetinostat, and we expect to have initial data from Phase II studies by the end of the year which will allow us to move quickly into a registration path."