21st Dec 2021 09:01
(Alliance News) - AstraZeneca PLC said Tuesday that its supplemental biologics license application for its myastenia gravis treatment, Ultomiris, has been accepted for priority review by the US Food & Drug Administration.
The Cambridge-based pharmaceutical and biotechnology company said the submission to US FDA was based on results from the phase three trial of Ultomiris in the treatment of myasthenia gravis. The trial showed efficacy as early as week one and sustained efficacy for 52 weeks, the company explained.
Myasthenia gravis is a rare, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness. AstraZeneca said the diagnosed prevalence of myasthenia gravis is estimated at 64,000 in the US.
Regulatory submissions for Ultomiris are also currently under review with health authorities in the European Union and Japan, AstraZeneca added.
Ultomiris is currently approved in the US, EU and Japan for treatment of adults with paroxysmal nocturnal haemoglobinuria, a rare blood cell disorder. It works by inhibiting the C5 protein in the complement cascade, a part of the body's immune system. When C5 is activated in an uncontrolled manner it can lead the body to attack its own healthy cells.
Those with myasthenia gravis also experience issues along the complement cascade due to the production of specific antibodies. These antibodies bind to the surface of cells and lead the immune system to attack a connection point between nerve cells and muscles known as the neuromuscular junction which leads to loss of muscle function.
Marc Dunoyer, chief executive of AstraZeneca's rare disease unit Alexion Pharmaceuticals, 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 myasthenia gravis. The phase three trial shows that Ultomiris may help a broader range of patients including those with milder symptoms or who are earlier in their treatment journey."
Soliris is another treatment developed by Alexion that also inhibits the C5 protein. It predominantly used for the treatment of paroxysmal nocturnal hemoglobinuria.
Shares in AstraZeneca were up 0.3% at 8,516.00 pence on Tuesday morning in London.
By Heather Rydings; [email protected]
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