UCB, a global biopharmaceutical company, received approval from the FDA for ZILBRYSQ (zilucoplan) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive.
 
ZILBRYSQ is the first once-daily subcutaneous, targeted peptide inhibitor of complement component 5 (C5 inhibitor)2. 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, ZILBRYSQ inhibits complement-mediated damage to the neuromuscular junction through its targeted mechanism of action. Unlike monoclonal antibody C5 inhibitors, as a peptide, ZILBRYSQ can be used concomitantly with intravenous immunoglobulin and plasma exchange, without the need for supplemental dosing.
 
The FDA approval is supported by safety and efficacy data from the Phase 3 RAISE study, a multi-center, randomized, double-blind, placebo-controlled study. Patients were randomized to receive daily subcutaneous injections of 0.3 mg/kg ZILBRYSQ or placebo for 12 weeks. The study demonstrated that ZILBRYSQ 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.
 
gMG is a rare, chronic, heterogeneous, unpredictable autoimmune disease characterized by dysfunction and damage at the neuromuscular junction (NMJ). Several factors are understood to be drivers of gMG disease pathology, including the complement cascade, immune cells and pathogenic Immunoglobulin G (IgG) autoantibodies.