New Zilbrysq delivery option OK’d by FDA for adults with myasthenia gravis
Autoinjector version of approved gMG therapy wins regulatory approval
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Beginning early next year, certain adults in the U.S. with generalized myasthenia gravis (gMG) will have a new option for receiving the approved gMG treatment Zilbrysq (zilucoplan): self-administering the drug with an autoinjector at home or having a caregiver give it to them using the device.
The U.S. Food and Drug Administration (FDA) has cleared an autoinjector version of Zilbrysq, a so-called complement inhibitor for people 18 and older with gMG who are positive for antibodies targeting the acetylcholine receptor (AChR) protein. The therapy, approved in the country since 2023, is given once daily via a subcutaneous, or under-the-skin, injection. Now, with appropriate training, it will soon be available to be administered at home by caregivers or patients themselves using this new delivery method.
According to UCB, the company that markets Zilbrysq, the new autoinjector device will be available to patients in early 2027. The company noted that the new formulation of Zilbrysq is the first and only FDA-approved option for gMG given daily with an autoinjector.
“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 daily use?” Kimberly Moran, PhD, senior vice president and head of U.S. rare diseases at UCB, said in a company press release announcing the FDA’s decision.
“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,” Moran said, adding that, “at UCB, we are committed to science and make it our point to continually help improve how people experience it.”
Zilbrysq is now available in prefilled syringes, which require a second person to physically stick the needle into the patient and push a plunger to get the medication into the body. Autoinjectors are designed to simplify the injection process by using a device that is placed on the skin and inserts a needle to inject the medication at the touch of a button. This can make treatment easier and more convenient, the developer noted.
Myasthenia gravis, or MG for short, is an autoimmune disease driven by self-reactive antibodies that interfere with communication from nerve to muscle cells. It leads to symptoms such as fatigue and muscle weakness. In contrast to ocular MG, in which the eyes and eyelids are affected, individuals with gMG typically experience weakness in a broader range of muscles throughout the body.
Antibodies targeting the AChR protein are the most common type of MG-driving antibody. When antibodies bind to AChR, they can activate a group of inflammatory proteins called the complement cascade, which helps drive disease activity.
Zilbrysq contains a small molecule that blocks complement activation, thereby limiting damage.
Trial participants highly satisfied with new Zilbrysq delivery option
The FDA’s approval of the autoinjector version of Zilbrysq was based largely on data from two clinical trials: the Phase 1 study DV0012 (NCT06511076) and the Phase 3b trial DV0013 (NCT06471361), both launched in 2024.
The Phase 1 study tested the autoinjector against the already-approved prefilled syringe in healthy volunteers. The two formulations were found to deliver equivalent levels of the active medication to the body, according to researchers.
The late-stage trial involved 31 people with AChR-related gMG who’d already been taking the prefilled-syringe version of Zilbrysq and switched to the autoinjector. Those results showed that the patients were able to make this switch and self-administer the autoinjector version. Moreover, the participants generally reported high satisfaction with the autoinjector version.
For a chronic condition like gMG, having different administration options allows patients to choose the approach that best fits their individual preferences and routines.
“From a medical standpoint, it was essential to confirm that this added administrative option didn’t come at the expense of safety and efficacy,” said Donatello Crocetta, MD, global head of medical affairs and chief medical officer at UCB. “The data supporting the autoinjector’s complete dose delivery show that this option continues to offer an effective treatment for patients.”
The FDA approval was also supported by a study that enrolled people with gMG along with caregivers and clinicians. Of the 100 participants, 90 successfully administered Zilbrysq’s autoinjector on their first attempt without training, per the developer.
“For a chronic condition like gMG, having different administration options allows patients to choose the approach that best fits their individual preferences and routines,” Crocetta said.
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