FDA gives priority review to generalized myasthenia gravis treatment

The FDA has given priority review to a biologic license application from UCB for rozanolixizumab, a treatment for adults with generalized myasthenia gravis, the biopharmaceutical company announced in a press release.

The FDA’s priority review follows recent validation from the European Medicines Agency (EMA) on the marketing authorization application for rozanolixizumab, a subcutaneous monoclonal antibody that targets the neonatal Fc receptor for adults who are anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibody positive.

Both FDA and EMA submissions are based on phase 3 study data, which showed that treatment with rozanolixizumab demonstrated clinically meaningful and significant improvements in myasthenia gravis outcomes, the release stated.

“People living with [myasthenia gravis] suffer from unpredictable, fluctuating and debilitating symptoms that have a huge impact on their lives, and there is a clear need for additional targeted treatments,” Charl van Zyl, executive vice president of neurology solutions at UCB, said in the release.

Rozanolixizumab significantly reduced myasthenia gravis activities of daily living scale scores from baseline to day 43, with a greater percentage of patients achieving at least a two-point improvement in scores compared with placebo, according to the release. Generalized myasthenia gravis is a chronic and unpredictable autoimmune disease in which pathogenic autoantibodies can impair synaptic transmission at the neuromuscular junction by targeting specific proteins on the post-synaptic membrane. This disrupts the nerves’ ability to stimulate the muscle.

In the United States, prevalence of generalized myasthenia gravis is estimated at 14 to 20 cases per 100,000 population, or approximately 36,000 to 60,000 cases. In Europe, the prevalence is estimated to be 10 per 100,000 people.

Feedback from the FDA and EMA is expected during the second quarter of 2023, UCB stated.