Wednesday, January 4, 2012

FDA grants orphan status for Halo's DMD drug candidate

By Michelle Lang

Halo Therapeutics LLC, a Newton biotech founded through a unique do-it-yourself drug development process, has won orphan drug designation from the U.S. Food and Drug Administration for HT-100, its treatment for Duchenne muscular dystrophy (DMD).

The orphan drug status gives Halo seven years of marketing exclusivity following approval of HT-100, as well as clinical trial funding support, tax credits linked to development costs and FDA user fee waivers. The designation is designed to promote rare disease therapeutics development.

HT-100, also known as halofuginone, addresses DMD by promoting healthy muscle fiber regeneration, the company said in a news release, to counter the disease’s muscle weakening progression. The neuromuscular disorder affects about one in 3,500 boys worldwide and generally is fatal by the time boys reach their twenties or thirties. Halofuginone is set to begin Phase 2 clinical trials in patients with DMD this year, Halo CEO Marc Blaustein said in a statement.

Halo Therapeutics is a unique business in that it was founded and funded by DMD patient advocacy groups Charley’s Fund and the Nash Avery Foundation, as well as industry veterans. The company is a spinout of virtual firm Dart Therapeutics LLC, which incorporated in Massachusetts in 2010, with former Genzyme SVP Eugene Williams as chairman.