RARE Daily

EC Expands Approval of Alexion’s Ultomiris to Include NMOSD

May 11, 2023

Rare Daily Staff

The European Commission granted expanded approval to Alexion’s Ultomiris as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with the rare autoimmune condition neuromyelitis optica spectrum disorder.

The approval is for anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). Approximately three-quarters of people with NMOSD are anti-AQP4 Ab+, meaning they produce antibodies that bind to the protein AQP4.

NMOSD is a rare and debilitating autoimmune disease that affects the central nervous system (CNS), including the spine and optic nerves. Most people living with NMOSD experience unpredictable relapses, characterized by a new onset of neurologic symptoms or worsening of existing neurologic symptoms, which tend to be severe and recurrent and may result in permanent disability. The diagnosed prevalence of adults with NMOSD in the EU is estimated at approximately 5,000.

Ultomiris is the first and only long-acting C5 complement inhibitor. It provides immediate, complete, and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks in adult patients, following a loading dose. It is approved to treat a number of other rare, autoimmune conditions.

The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the CHAMPION-NMOSD phase 3 trial, which were published online in the Annals of Neurology. In the trial, Ultomiris was compared to an external placebo arm from the pivotal Soliris PREVENT clinical trial.

Ultomiris met the primary endpoint of time to first on-trial relapse as confirmed by an independent adjudication committee. Zero relapses were observed among Ultomiris patients with a median treatment duration of 73 weeks (relapse risk reduction: 98.6 percent, hazard ratio and continuing through a median duration of 90 weeks.

Overall, the safety and tolerability of Ultomiris in the CHAMPION-NMOSD trial were consistent with previous clinical studies and real-world use, and no new safety signals were observed. The most common adverse events (AEs) were COVID-19, headache, back pain, arthralgia and urinary tract infection. All cases of COVID-19 were non-serious and considered to be unrelated to Ultomiris.

“Effective NMOSD management hinges on reducing relapses—even a single relapse may cause irreversible disabilities leaving patients unable to move, speak or care for themselves,” said Orhan Aktas, professor at the Department of Neurology, Medical Faculty at Heinrich-Heine-University, Düsseldorf, Germany. “This approval in Europe will offer patients and physicians a new, long-acting treatment with unmatched relapse risk reduction, as Ultomiris showed zero relapses in its pivotal trial with dosing every eight weeks.”

Ultomiris is approved in the U.S., EU and Japan for the treatment of certain adults with generalized myasthenia gravis.

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