Ultomiris, one of the major drugs marketed by New Haven’s Alexion Rare Disease, was approved for use in adults with a common form of myasthenia gravis, parent company AstraZeneca reported last week.
The drug, also known as ravulizumab-cwvz, was approved for treatment of adult patients with the chronic autoimmune disorder who are anti-acetylcholine receptor (AChR) antibody-positive, representing 80% of people living with the disease.
The FDA gave its OK after positive results were reported in a Phase 3 trial which found that Ultomiris improved the daily life of patients when compared to a placebo. The drug is the first of its type approved for treatment of myasthenia gravis, according to AstraZeneca.
Patients take the drug once every eight weeks, offering greater convenience in addition to its positive effects, the drugmaker said.Â
About 90,000 people in the U.S. live with myasthenia gravis (MG), which can lead to a debilitating loss of muscle function and severe weakness.
Myasthenia Gravis Foundation of America CEO Samantha Masterson said, “With the approval of Ultomiris, we’re excited that MG patients now have another option to consider as part of their personalized treatment strategies that may offer more convenience and improve muscle weakness.”
Ultomiris, along with rare-disease drug Soliris, were the major assets of Alexion when it was acquired by AstraZeneca in July 2021 in a deal valued at $39 billion.Â
AstraZeneca scrapped a clinical trial for Ultomiris in August 2021 after the drug failed to show effectiveness against the deadly neuromuscular disease amyotrophic lateral sclerosis, or ALS.
Ultomiris has been approved in the U.S., Europe and Japan for the treatment of a subset of patients with rare diseases paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, and is being assessed for treatment of additional ailments in hematology and neurology.
Contact Liese Klein at lklein@newhavenbiz.com.
