FDA approves ZILBRYSQ autoinjector self-administration for adults living with generalized myasthenia gravis – UCB

UCB announced that the FDA has approved the first self-administered once-daily, autoinjector administration option for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive with ZILBRYSQ (zilucoplan).

The autoinjector is designed to help simplify self-administration of ZILBRYSQ, offering patients an another administration option and a more convenient treatment experience.

“This approval is the answer to a question on which we’ve been relentlessly focused: how do we continue to improve an already effective therapy for use every single day?” said Kimberly Moran, PhD, SVP & Head, U.S. Rare Diseases at UCB. “As the first treatment to be approved as a once-daily autoinjector option for adults living with gMG in the U.S., ZILBRYSQ now provides the same therapy via another administration option. At UCB, we are committed to science and make it our point to continually help improve how people experience it.”

The ZILBRYSQ autoinjector is designed to automatically deliver a pre-defined dose when activated against the skin. Prior to the autoinjector’s approval, ZILBRYSQ was available as a self-administered prefilled syringe (PFS). Patients or their care partners will now have another portable treatment solution.

“For a chronic condition like gMG, having different administration options allows patients to choose the approach that best fits their individual preferences and routines,” said Donatello Crocetta, Global Head of Medical Affairs and Chief Medical Officer at UCB. “From a medical standpoint, it was essential to confirm that this added administrative option didn’t come at the expense of safety and efficacy. The data supporting the autoinjector’s complete dose delivery show that this option continues to offer effective treatment for patients.”

The approval of the ZILBRYSQ autoinjector for self-administration is supported by data from the following studies:

DV0012 – a Phase 1, open-label, single-center, two-period crossover study in healthy adult volunteers (n=14) which established bioequivalence of ZILBRYSQ delivered via an autoinjector compared with a PFS.

DV0013 – a Phase 3b open-label, multi-center study in adult patients (n=31) with gMG self-administering ZILBRYSQ via autoinjector once daily. Complete dose delivery was achieved for 99.8% (n=449) of autoinjector administrations. Patients reported high satisfaction with self-injection of ZILBRYSQ using the autoinjector device. Self-administration was generally well tolerated. The most common TEAE was injection-site pain (n=3; 9.7%).

A human factors validation study of 100 participants demonstrating that 90% successfully completed an injection with the ZILBRYSQ autoinjector on their first attempt without training.

UCB expects the autoinjector to be available commercially in Q1 2027.