The approval was based on the safety and efficacy data from the RAISE phase 3 study in which Zilbrysq delivered fast, consistent, and statistically significant benefits in different patient-and-clinician reported outcomes at week 12
UCB’s building image. (Credit: UCB S.A.)
Biopharmaceutical company UCB said that the US Food and Drug Administration (FDA) has approved Zilbrysq (zilucoplan) for the treatment of generalised myasthenia gravis (gMG) in adult patients.
Zilbrysq is a once-daily, subcutaneous (SC), self-administered targeted peptide inhibitor of complement component 5 (C5 inhibitor).
The FDA has approved zilucoplan for gMG adults who are anti-acetylcholine receptor (AChR) antibody positive.
The approval was based on the safety and efficacy data from the RAISE phase 3 study, whose results were published in The Lancet Neurology in May.
In the trial, the patients were randomised in a 1:1 ratio to get daily SC injections of 0.3 mg/kg zilucoplan or placebo for 12 weeks.
According to the results, Zilbrysq delivered fast, consistent, and statistically significant benefits in different patient-and-clinician-reported outcomes at week 12 in patients with mild-to-severe anti-AChR-antibody positive gMG.
Furthermore, the most common adverse reactions were upper respiratory tract infection, injection site reactions, and diarrhoea.
UCB CEO Jean-Christophe Tellier said: “Following the FDA approval and strong momentum with our FcRn inhibitor rozanolixizumab-noli and with our tailored patient support services and commitment to widespread access, I am confident that UCB is the only company with a portfolio of two targeted therapies with different mechanisms of action and the experience to deliver truly individualised transformational patient value to people living with this often debilitating rare disease.
“We would like to extend our thanks to the patients, care partners, and investigators who participated in the RAISE study, and to our employees and collaborators, whose dedication and commitment to the gMG community made this important milestone possible.”
RAISE is a multi-centre, late-stage, randomised, double-blind, placebo-controlled study. It had a goal to evaluate the efficacy, safety profile, and tolerability of zilucoplan in adult patients with AChR antibody-positive gMG.
The primary endpoint was defined as the change from baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score.
This approval complements the biopharmaceutical firm’s neonatal Fc receptor (FcRn) blocker Rystiggo, which was cleared by the FDA under the Priority Review designation.