A novel drug shows promise as a potential options for patients with myasthenia gravis (MG). It gained a designated priority review of the United States Food and Drug Administration (FDA), according to a press release by UCB.
“Patients living with MG may experience high disease and treatment burden resulting in a significant impact on their daily lives. If approved, rozanolixizumab has the potential to address unmet needs of gMG patients,” highlighted Iris Loew-Friedrich, chief medical officer and executive vice president of UCB.
Rozanolixizumab is a new drug that recently received the priority review designation after the company applied for the Biologic License Application of the FDA in November 2022. This drug had already achieved other important recognitions, such as the European Medicines Agency (EMA) validation in 2020 and the orphan drug designation of the FDA in 2019.
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This approval comes as the next step after the completion of phase 3, a double-blind, randomized, multicenter clinical trial named MycarinG. This study included 200 patients that were randomized into either 1 of the 3 groups: the initial 1 received rozanolixizumab at a dose of 10 mg/kg, the second group received a dose of 7 mg/kg, while the third group received a placebo.
The trial evidenced a statistically significant improvement with positive clinical outcomes among those who received the drug, meeting the primary endpoint of decreasing the MG-Activities of Daily Living (MG-ADL) scale on day 43 of follow-up. All patients had generalized MG with either anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibodies.
Moreover, the drug exhibited a promising safety profile, resulting in acceptable adverse events with a frequency of 81.3% in the 10 mg/kg and 82.6% in the 7 mg/kg dose groups. The placebo group experienced adverse events in 67.2% of the cases.
This medication is a humanized monoclonal antibody that is specific for the neonatal Fc receptor (FcRn). Rozanolixizumab binds to its target with high affinity, promoting a faster decrease in the IgG autoantibodies by inhibiting the IgG contact with FcRn.
UCB announces rozanolixizumab BLA for the treatment of generalized myasthenia gravis filed with U.S. FDA anddesignated for Priority Review. News release. UCB; January 06, 2023.