Tuesday, April 16, 2024

Approval Granted for Innovative Generalized Myasthenia Gravis Treatment

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The European Commission (EC) has given its authorization for ZILBRYSQ® (zilucoplan), a once-daily subcutaneous (SC) C5 complement inhibitor, making it the initially targeted peptide inhibitor of complement component 5 (C5) approved for treating generalized myasthenia gravis (gMG). This approval marks a significant step as it is the sole C5 inhibitor sanctioned for self-administration among adult patients with this condition who are AChR antibody-positive.

Zilucoplan endorsed as an adjunct to standard therapy, targets adults who test positive for anti-acetylcholine receptor (AChR) antibodies and suffer from this rare autoimmune disease, as indicated by the European Medicines Agency (EMA).

Zilucoplan’s Rapid Impact on Myasthenia Gravis

The authorization was granted based on results from the RAISE Phase III study, revealing substantial and consistent enhancements in outcomes by week 12, the study’s completion, attributable to zilucoplan’s rapid and significant impact.

Patients enrolled in the RAISE trial received daily subcutaneous injections of 0.3mg/kg zilucoplan or a placebo over 12 weeks. Following the completion of the RAISE trial, patients had the option to join the open-label RAISE-XT extension study, according to UCB.

Unlike monoclonal antibody C5 inhibitors, zilucoplan, being a peptide-based small molecule, permits concurrent usage with intravenous immunoglobulin and plasma exchange, eliminating the necessity for supplemental dosing based on Phase III trial findings.

Signs and Symptoms of Myasthenia Gravis

EC Approval of Zilucoplan and Advances in Rozanolixizumab

UCB’s CEO, Jean-Christophe Tellier, highlighted that alongside the EC’s approval of zilucoplan, other regulatory advancements in treating adults with gMG include a favorable European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) opinion for UCB’s FcRn blocker rozanolixizumab and approvals for both zilucoplan and rozanolixizumab in the US and Japan.

Rozanolixizumab, a humanized IgG4 monoclonal antibody, works by reducing circulating IgG levels through the inhibition of FcRn and Immunoglobulin G (IgG) interaction. This mechanism effectively decreases the concentration of pathogenic IgG autoantibodies in the body, providing an alternative treatment approach detailed in a Lancet paper.


Resource: European Pharmaceutical Review, December 05, 2023

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