UCB, a global biopharmaceutical company, announced on October 17th that ZILBRYSQ® (zilucoplan) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive.
“For people with gMG, the unpredictable nature of the severity and frequency of symptoms can be debilitating and can have a substantial impact on many aspects of their day-to-day lives. In addition to muscle weakness, people living with gMG experience fatigue, affecting their overall quality of life,” said James F. Howard, MD, Distinguished Professor of Neuromuscular Disease, Professor of Neurology, Medicine and Allied Health, The University of North Carolina at Chapel Hill School of Medicine and lead investigator in the RAISE clinical trial of zilucoplan. “Zilucoplan demonstrated rapid improvements in gMG symptoms at Week 12, with differences seen as early as one week, and provides a new treatment option for a broad population of AChR antibody-positive gMG patients. Zilucoplan is designed for continued daily use.”
Zilucoplan is the first once-daily subcutaneous (SC), targeted peptide inhibitor of complement component 5 (C5 inhibitor). It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.
As a complement C5 inhibitor, zilucoplan inhibits complement-mediated damage to the neuromuscular junction through its targeted mechanism of action. Benefits of self-administered treatment compared with intravenously administered treatments can include reduced traveling time to and from hospitals, decreased interference with work obligations, and increased independence. Unlike monoclonal antibody C5 inhibitors, as a peptide, zilucoplan can be used concomitantly with intravenous immunoglobulin and plasma exchange, without the need for supplemental dosing.
The FDA approval of zilucoplan is supported by safety and efficacy data from the RAISE study (NCT04115293), published in The Lancet Neurology in May 2023. The RAISE study was a multi-center, phase 3, randomized, double-blind, placebo-controlled study to assess the efficacy, safety profile, and tolerability of zilucoplan in adult patients with anti-acetylcholine receptor (AChR) antibody-positive gMG. Patients were randomized in a 1:1 ratio to receive daily subcutaneous (SC) injections of 0.3 mg/kg zilucoplan or placebo for 12 weeks. The study demonstrated that zilucoplan delivered rapid, consistent, and statistically significant benefits in different patient-and-clinician reported outcomes at week 12 in a broad population of adult patients with mild-to-severe anti-AChR-antibody positive gMG. The most common adverse reactions (≥10%) in patients with gMG were injection site reactions, upper respiratory tract infection, and diarrhea.
To read the full press release from UCB, click here.