Friedreich’s ataxia (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, which commonly progresses to motor incapacitation and wheelchair reliance. There are no currently approved therapies for the treatment of FA.
"Orphan drug designation serves as an important milestone for our company as it recognizes the promise of omaveloxolone as a potential new treatment for FA. In light of the recent, encouraging clinical data, we are hopeful that omaveloxolone will be the first therapy approved for patients with FA," said
Earlier this month, Reata released positive data from Part 1 of the Company’s Phase 2 trial (MOXIe) of omaveloxolone for the treatment of Friedreich’s ataxia. The trial demonstrated that in FA patients, omaveloxolone induced Nrf2, which is suppressed in FA patients, and this was associated with improvements in mitochondrial and neurological function. Dose-dependent and time-dependent effects on the modified Friedreich’s Ataxia Rating Scale (mFARS) were observed at the pharmacodynamically-active doses. The Company expects to initiate Part 2 of MOXIe during the second half of 2017.
Orphan drug status is granted to treatments for diseases that affect fewer than 200,000 people in the U.S. and provides specific incentives to therapies intended for the treatment, diagnosis, or prevention of rare diseases. Such designation will provide Reata with certain development incentives, including tax credits for clinical testing, exemption from a prescription drug user fee, and seven years of market exclusivity.
About Friedreich’s Ataxia
FA is a rare, degenerative, life-shortening neuro-muscular disorder that affects children and adults and involves the loss of strength and coordination usually leading to wheelchair use; diminished vision, hearing and speech; scoliosis (curvature of the spine); increased risk of diabetes; and a life-threatening heart condition. Currently, there are no
Reata is a clinical-stage biopharmaceutical company that develops novel therapeutics for patients with serious or life-threatening diseases by targeting molecular pathways involved in the regulation of cellular metabolism and inflammation. Reata’s two most advanced clinical candidates, bardoxolone methyl and omaveloxolone, target the important transcription factor Nrf2 to restore mitochondrial function, reduce oxidative stress, and resolve inflammation.
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