Omaveloxolone, a drug put out by Reata Pharmaceuticals, has recently received an Orphan drug designation for Friedreich’s ataxia from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA). This means that Reata now has the Orphan drug designation for Omaveloxolone from both the FDA (US) and the EMA (EU). You can find the press release here about this new designation.

The drug displayed positive effects in an in vitro study (read the press release here and has been further supported by the first part of a 2-part international, multi-centre, randomized, double-blind, placebo-controlled phase 2 trial, referred to as MOXIe. Results from part one of the study have indicated dose and time dependent improvements in those treated with omaveloxolone.

Reata is currently in the process of enrolling about 100 patients with Friedreich's ataxia for part 2 of the MOXIe trial; results are expected in second half of 2019. This drug is the same as the one currently in trials around the world including at the London Ataxia Centre. If you wish to take part you can find more information here.

Posted on 13/07/2018