The EMA’s decision follows orphan drug designation from the U.S. Food and Drug Administration granted in June 2017.
These designations are expected to provide regulatory support and financial benefits, to accelerate the clinical development and review of omaveloxolone, and to ensure marketing exclusivity for a period of time upon regulatory approval.
“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,” Warren Huff, Reata’s president and CEO, said in a press release.
The company is currently recruiting an estimated 100 FA patients for the next part of a Phase 2 clinical trial (NCT02255435) on omaveloxolone, called MOXIe.
FA is a rare neurodegenerative disorder affecting approximately 6,000 children and adults in the U.S. and 22,000 people worldwide. It is caused by deficient production of frataxin, an essential protein for the normal functioning of mitochondria, which provide energy to cells.
This leads to mitochondrial dysfunction and impaired energy production, low levels of antioxidant molecules that protect cells from oxidative stress, and chronic inflammation, which ultimately results in progressive neurodegeneration.
While many of the symptoms and complications of FA can be eased to help patients maintain optimal functioning for as long as possible, there is no approved treatment to reduce or prevent disease progression.
Omaveloxolone, being developed by Reata in collaboration with AbbVie, is a small molecule designed to specifically target and activate Nrf2 — a protein whose function is impaired in FA patients.
Nrf2 is involved in restoring mitochondrial function, improving the levels of antioxidant agents that reduce oxidative stress and blocking inflammation, indicating that its activation could potentially help treat FA patients.
The safety, effectiveness, and pharmacodynamics — the therapy’s effects and mechanism of action — of oral omaveloxolone are being evaluated in the two-part international, randomized, double-blind, placebo-controlled Phase 2 MOXIe trial.
Results reported last year from the dose-escalation first part of the study showed that omaveloxolone induced dose- and time-dependent improvements in mitochondrial and nerve cell function.
The trial’s second part, which is currently enrolling, is a parallel-group study designed to evaluate the safety and effectiveness of a daily 150 mg dose of omaveloxolone in FA patients, who will be randomized to receive either omaveloxolone or a placebo. Results from part two are expected in the second half of 2019.
The study is sponsored by Reata, in collaboration with AbbVie and the Friedreich’s Ataxia Research Alliance.
Those who complete the MOXIe study will be eligible to enter an extension trial to assess the long-term safety and tolerability of omaveloxolone, where all participants will receive open-label omaveloxolone at 150 mg once daily.