Ultomiris (Ravulizumab-cwvz)

Ultomiris (ravulizumab-cwvz) is a treatment being developed by Alexion Pharmaceuticals for amyotrophic lateral sclerosis (ALS). The company submitted an investigational new drug application to the U.S. Food and Drug Administration (FDA) in 2019.

Ultomiris already has been approved by the FDA as a therapy for atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria.

How does Ultomiris work?

ALS is a progressive neurological disorder characterized by progressive neurodegeneration — loss of nerve cells — in the brain and spinal cord. When the nerve cells die, the brain cannot control muscle movement, which causes progressive paralysis, among other symptoms.

Ultomiris contains an antibody that is injected into the bloodstream and that inhibits a protein called C5, a member of the complement system. The complement system is part of the immune system and is thought to be implicated in neurodegeneration in ALS.

Under normal conditions, the complement system acts as part of the innate immune system to enhance the function of antibodies and the immune cells that recognize threats like infections. The complement system is activated at a low level at all times. Under some circumstances, however, an ALS-triggering event, for example, such as increased inflammation around nerve cells in the brain, the complement system overreacts.

By inhibiting C5, Ultomiris interrupts the overreaction of the complement system. In that way, the therapy may be able to slow disease progression in ALS.

Ultomiris in clinical trials

Alexion is planning a Phase 3, randomized, double-blind, placebo-controlled multicenter clinical trial called CHAMPION-ALS to evaluate the safety and effectiveness of Ultomiris in patients with ALS. The study intends to recruit 350 adults with either sporadic or familial ALS. To be eligible for the study, patients’ symptoms must have started in the past three years prior to the trial. Patients also must have a slow vital capacity (SVC, a measure of respiratory function) of 65%, and not be dependent upon respiratory support.

Participants will be assigned randomly at a 2:1 ratio to receive Ultomiris or placebo every eight weeks following an initial loading dose. Patients can continue their existing treatment in addition to Ultomiris or placebo. After 50 weeks, all patients will receive Ultomiris in a two-year open-label extension of the study. The study will take place at approximately 90 clinical trial sites across North America, Europe, and Asia-Pacific. The primary goal for the study will be change from baseline in ALS functional rating scale-revised (ALSFRS-R) score. Secondary goals nclude ventilation assistance-free survival (VAFS), respiratory capacity, muscle strength, and safety.


Last updated: Jan. 21, 2020


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