New Weekly Therapy Recommended for Myasthenia Gravis in EU

Adults living with generalized myasthenia gravis (gMG) in Europe could soon have access to a new at-home weekly treatment option. The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for Klygefa (gefurulimab, Alexion/AstraZeneca) as an add-on to standard therapy for adults with gMG who are anti-acetylcholine receptor (AChR) antibody-positive.gMG is a rare autoimmune disorder in which antibodies attack the neuromuscular junction, disrupting communication between nerves and muscles. The condition causes fluctuating, often debilitating muscle weakness that can progress to impaired swallowing, extreme fatigue, and, in severe cases, respiratory failure. Roughly 85% of people with gMG are AChR antibody-positive. How It Works Klygefa is a 300-mg solution for injection in a prefilled pen or prefilled syringe designed for subcutaneous injection. It works by binding to complement protein C5, blocking its cleavage into the pro-inflammatory fragments C5a and C5b. This prevents pathogenic activation of the terminal complement cascade, which contributes to disease in AChR-positive gMG. Gefurulimab also binds to serum albumin, which extends its half-life and allows weekly dosing. Clinical Evidence Supporting evidence included the phase 3 PREVAIL trial, a placebo-controlled study of 260 adults with AChR antibody-positive gMG treated with once-weekly subcutaneous gefurulimab or placebo for 26 weeks. Gefurulimab significantly improved Myasthenia Gravis Activities of Daily Living scores at week 26 vs placebo; benefits were seen within 1 week of treatment and were sustained through week 26. Adverse event rates were similar between the study groups.The CHMP summary states that Klygefa reduced disease severity, improved function, and improved quality of life. The most common side effects are injection-site reactions, back pain, arthralgia, muscle spasms, myalgia, nausea, and vomiting.Next Steps The CHMP opinion now goes to the European Commission, which typically issues a final, legally binding decision on EU-wide marketing authorization within about 2 months. If granted, Klygefa should be used under the guidance of healthcare professionals experienced in managing patients with neuromuscular disorders. Detailed recommendations for use will be described in the Summary of Product Characteristics, which will be published on the EMA website in all official European Union languages once approval is confirmed.