Astra Zeneca said Tuesday that Ultomiris regulatory submission has been accepted under FDA Priority Review in the US for adults with generalised myasthenia gravis, based on positive Phase III trial in which Ultomiris significantly improved functional activities as measured by Myasthenia Gravis-Activities of Daily Living Profile.
The FDA set a Prescription Drug User Fee Act date during the second quarter of 2022, following use of a rare disease priority review voucher by Alexion, AstraZeneca’s Rare Disease group.
gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness. The diagnosed prevalence of gMG in the US is estimated at 64,000.
Marc Dunoyer, Chief Executive Officer, Alexion, said: “Soliris was the first new treatment approved for this devastating disease in approximately 60 years, and this filing for Ultomiris demonstrates Alexion’s continued commitment to improve outcomes for patients living with gMG. The Phase III trial shows that Ultomiris may help a broader range of patients including those with milder symptoms or who are earlier in their treatment journey.”
The sBLA submission in the US is based on results from the Phase III trial of Ultomiris in gMG, which were announced by Alexion in July 2021, and showed efficacy as early as Week 1 and sustained for 52 weeks (26 weeks randomised controlled period + 26 weeks of open-label extension). In the trial, the safety profile of Ultomiris was consistent with that observed in Phase III trials of Ultomiris in paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS).
Regulatory submissions for Ultomiris for the treatment of gMG are also currently under review with health authorities in the European Union (EU) and Japan.