Wednesday, July 11, 2018
Reata Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, today announced that the European Commission has granted orphan drug designation for omaveloxolone for the treatment of Friedreich’s ataxia (FA), based on the positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA).
FA is an inherited, debilitating, and degenerative neuromuscular disorder that is typically diagnosed during adolescence and can ultimately lead to early death. Patients with FA experience progressive loss of coordination, muscle weakness, and fatigue that commonly progresses to motor incapacitation and wheelchair reliance. FA affects approximately 6,000 children and adults in the United States and 22,000 globally. Currently, there are no drugs approved for the treatment of FA.
Last year, Reata reported results from part 1 of MOXIe, a two-part, international, multi-center, randomized, double-blind, placebo-controlled Phase 2 trial studying the safety and efficacy of omaveloxolone in patients with FA. Treatment of FA patients with omaveloxolone produced dose- and time-dependent improvements in their modified Friedreich’s Ataxia Rating Scale (mFARS) scores, which are a measure of the neurologic function of FA patients. Reata is currently enrolling approximately 100 FA patients in the registrational part 2 portion of MOXIe and expects to have results in the second half of 2019.
“Orphan drug designation from the EMA is an important recognition of the potential for omaveloxolone to become the first approved therapy for patients affected by this devastating disease,” said Warren Huff, Reata’s President and Chief Executive Officer.
In Europe, orphan drug designation is granted to therapies intended for the treatment of life-threatening or chronically debilitating diseases that affect no more than five in 10,000 people in the European Union and for which no satisfactory treatments are available, or where the new therapy has the potential to be a significant benefit to those affected by the disease. Orphan designation provides specific financial and regulatory incentives, including reduced fees, protocol assistance, access to the centralized authorization procedure, and ten years of market exclusivity once the drug is approved.