The sBLA submission was based on positive results from the Phase 3 trial, in which Ultomiris significantly improved functional activities
US FDA grants Priority Review for Ultomiris. (Credit: Business Wire.)
The US Food and Drug Administration (FDA) has accepted AstraZeneca’s supplemental Biologics License Application (sBLA), and granted priority review for Ultomiris (ravulizumab-cwvz) to treat generalised myasthenia gravis (gMG).
The US agency has finalised as Prescription Drug User Fee Act date in the second quarter of 2022, after AstraZeneca’s Rare Disease group, Alexion, used a priority review voucher.
Ultomiris is a long-acting therapeutic that works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system.
According to the company, Ultomiris is the first and only long-acting C5 complement inhibitor, designed to provide immediate, complete, and sustained complement inhibition.
Alexion CEO Marc Dunoyer 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 company’s sBLA is based on results from the Phase 3 trial in 175 patients, across North America, Europe, Asia-Pacific and Japan.
In the study, Ultomiris showed efficacy within the first week and sustained for 52 weeks, compared to placebo.
Its safety profile was consistent with that observed in Phase 3 trials in paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS).
Ultomiris was previously approved in the US for the treatment of PNH and aHUS in adults and children, along with the EU and Japan.
Furthermore, the company has also filed regulatory submissions with health authorities in the European Union (EU) and Japan, which are currently under review.
Generalised myasthenia gravis is a rare, debilitating, chronic, autoimmune neuromuscular disease characterised by severe muscle weakness.
The autoimmune disorder causes inflammation that damages the connection between nerve cells and the muscles they control, also called neuromuscular junction (NMJ).
The damage leads to interruption of communication between the brain and muscles, causing loss of muscle function and severe weakness.