FDA accepts NDA for generalized myasthenia gravis treatment
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The FDA has accepted a new drug application for zilucoplan, an investigational treatment for generalized myasthenia gravis in adults who are acetylcholine receptor antibody positive, UCB announced in a released statement.
According to the release, zilucoplan is a subcutaneous, self-administered peptide inhibitor of complement component 5 (C5) that inhibits key components in the underlying pathophysiology of generalized myasthenia gravis (gMG).
The FDA in 2019 granted orphan drug designation for zilucoplan for the treatment of myasthenia gravis, but it is not currently approved for use in any indication by any regulatory authority globally.
“People living with gMG experience high treatment burden, on top of the debilitating impact of the condition, and there is a clear need for additional targeted treatments to support the gMG community,” Charl van Zyl, executive vice president of neurology solutions at UCB, said in the release.
The acceptance of the NDA follows the recent European Medicines Agency (EMA) validation of the marketing authorization application (MAA) for zilucoplan in adults with acetylcholine receptor antibody positive gMG who require treatment in addition to steroids and nonsteroidal immunosuppressants. Orphan designation was granted for zilucoplan in 2022 by the European Commission.
“The acceptance of the NDA by the FDA, as well as the acceptance of the MAA by the EMA, brings us one step further on our journey towards approval for this medicine,” van Zyl said. “We look forward to working with the FDA and EMA to help bring this important new treatment option to patients.”
T he NDA and MAA are based on data from a phase 3 trial, which at week 12 showed that patients treat ed with zilucoplan (0.3 mg/kg daily) had clinically meaningful and statistically significant improvement in gMG-specific outcomes compared with those who received placebo, the company stated.
The study met its primary endpoints, with zilucoplan demonstrating a placebo-corrected mean improvement of 2.09 points in the Myasthenia Gravis Activities of Daily Living score at week 12. Zilucoplan also had a favorable safety and tolerability profile, with similar rates of adverse events (76.7%) compared with placebo (70.5%).