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Practical NeurologyFDA Approves Zilbrysq as First Once-Daily Subcutaneous C5 Inhibitor for Generalized Myasthenia Gravis

The approval could be a new dawn in the treatment of gMG

The US Food and Drug Administration (FDA) has given its approval for Zilbrysq (zilucoplan; UCB, Atlanta, GA), a treatment for generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor antibody positive (anti-AChR Ab+). This marks a significant milestone as Zilbrysq is the first once-daily subcutaneous, targeted complement component 5 (C5) inhibitor for gMG that is self-administered.

Key Points

  • Zilbrysq is the first approved once-daily subcutaneous, targeted complement component 5 (C5) inhibitor for gMG.
  • In anti-AChR Ab+ gMG, complement C5 mediates damage to the neuromuscular junction (NMJ), leading to skeletal muscle weakness.
  • The FDA approval is based on results from the RAISE study (NCT04115293), which were published in The Lancet Neurology.
  • RAISE was a phase 3 multicenter, randomized, double-blind, placebo-controlled study that assessed the efficacy, safety, and tolerability of Zilbrysq as a treatment for 174 adult participants with a diagnosis of anti-AChR Ab+ gMG.
  • The participants were randomized 1:1 to receive either .3 mg/kg Zilbrysq or placebo as a once-daily subcutaneous injection.
  • The primary outcome was change from baseline in participants’ total score on the Myasthenia Gravis–Activities of Daily Living (MG-ADL) scale.

“Until now, people living with gMG have only had access to C5 therapy intravenously, which can be inconvenient and time consuming. Now, with the option of ZILBRYSQ, a self-administered once-daily, subcutaneous targeted complement C5 inhibitor, we hope a broad population of mild-to-severe adult patients with AChR-antibody-positive gMG will be able to have greater independence.”
— Iris Loew-Friedrich, Executive Vice-President and Chief Medical Officer at UCB

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