UCB announced that ZILBRYSQ®? (zilucoplan) is now available in the U.S. for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. ZILBRYSQ was approved by the U.S. Food and Drug Administration (FDA) on October 17, 2023.

ZILBRYSQ is available by prescription as a ready-to-use pre-filled syringe that is a once-daily administration. Healthcare providers who prescribe ZILBRYSQ must enroll in the ZILBRYSQ REMS because of the risk of serious meningococcal infections. Myasthenia gravis is a rare, chronic, autoimmune neuromuscular disease, which is characterized by muscle weakness that worsens after periods of activity and improves after periods of rest.

In the U.S., there are 35,000 to 60,000 people living with myasthenia gravis who experience a variety of symptoms, including droopingeyelids, double vision, and difficulty in swallowing, chewing, and talking. Exacerbations are unpredictable and are characterized by potentially severe symptoms affecting activities of daily living. Now that ZILBRYSQ is approved and commercially available, the ONWARD??

program will be in place to provide further care and support for those navigating the complexities of this disease. With UCB's two FDA-approved targeted treatment options, which have different mechanisms of action and methods of administration, UCB will continue to deliver on commitment towards differentiated solutions for this community where there are great unmet needs. Based on the differentiated value of ZILBRYSQ as the first and only FDA-approved self-administered C5 complement inhibitor for AChR+ adults, UCB aimed to price ZILBRYSQ to be the lowest cost complement inhibitor treatment for the U.S. gMG population overall.

ONWARD, a personalized patient support program, will be available to eligible patients and caregivers to provide support throughout their prescribed treatment with medicines within the UCB Rare Disease Portfolio, including ZILBRYSQ and RYSTIGGO. Through ONWARD, eligible patients will have access to important resources, including a dedicated Care Coordinator+ who will provide personalized support, tools, and resources to get started on treatment. One are encouraged to report negative side effects of prescription drugs to the FDA.

Visit or call 1-800-FDA-1088. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans.

The length of the timing to complete clinical trials and to get regulatory approval for product candidates in humans. The size of the timing to complete clinical trial is expected to be in the U.S. and RYSTIGGO®?.