RARE Daily

FDA Approves Alexion Rare Diseases’ Ultomiris to Treat Adults with NMOSD

March 25, 2024

Rare Daily Staff

The U.S. Food and Drug Administration approved Alexion Rare Diseases’ supplemental new drug application for Ultomiris for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder.

The approval was based on positive results from the CHAMPION-NMOSD phase 3 trial, which were published 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.

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 were COVID-19, headache, back pain, arthralgia, and urinary tract infection.

Neuromyelitis optica spectrum disorder (NMOSD) is a rare and debilitating autoimmune disease that affects the central nervous system, 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 U.S. is estimated at approximately 6,000. Approximately three-quarters of people with NMOSD are anti-AQP4 Ab+, meaning they produce antibodies that bind to a specific protein, aquaporin-4 (AQP4).5,12 This binding can inappropriately activate the complement system, which is part of the immune system and is essential to the body’s defense against infection, to destroy cells in the optic nerve, spinal cord and brain.

It most commonly affects women and begins in the mid-30s. People with NMOSD may experience vision problems, intense pain, loss of bladder/bowel function, abnormal skin sensations (e.g., tingling, prickling or sensitivity to heat/cold) and impact on coordination and/or movement. Most people living with NMOSD experience unpredictable relapses, also known as attacks. Each relapse can result in cumulative disability including vision loss, paralysis, and sometimes premature death. NMOSD is a distinct disease from other CNS diseases, including multiple sclerosis. The journey to diagnosis can be long, with the disease sometimes misdiagnosed.

Ultomiris is the first and only long-acting C5 complement inhibitor that offers patients with AQP4 Ab+ NMOSD the potential to live relapse-free. 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.

“Building on the established efficacy of C5 inhibition for people living with AQP4 Ab+ NMOSD, we are proud to deliver a transformative, long-acting treatment option that has the potential to eliminate relapses with a convenient dosing schedule every eight weeks,” said Marc Dunoyer, CEO at Alexion.

Ultomiris is already approved for certain adults with NMOSD in Japan and the European Union. Ultomiris is also approved in the U.S., EU, Japan and other countries for the treatment of certain adults with generalized myasthenia gravis (gMG) and for the treatment of certain adults with paroxysmal nocturnal hemoglobinuria (PNH), and for certain children with PNH in the U.S. and EU. Additionally, Ultomiris is approved in the U.S., EU, Japan and other countries for certain adults and children with atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy (aHUS).

Photo: Marc Dunoyer, CEO at Alexion Rare Diseases

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