BLA Priority Review for Generalized Myasthenia Gravis Treatment Submitted to FDA

A Biologic License Application (BLA) designated Priority Review was submitted to the Food and Drug Administration (FDA) for rozanolixizumab (UCB7665; UCB, Atlanta, GA) as treatment for adults with generalized myasthenia gravis (gMG) who are anti-acetycholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive (Ab+). The application includes results from a phase 3 study which demonstrated improvements in various outcomes, including activities of daily living, disease severity, and symptoms, and coincides with a marketing authorization application submitted to the European Medicines Agency for rozanolixizumab. 

In the phase 3 MycarinG study (NCT03971422), rozanolixizumab demonstrated statistically significant and clinically meaningful improvements in MG-specific outcomes in patients with MuSK-Ab+ gMG that were consistent with results in acetylcholine receptor antibody positive (AChR-Ab+) gMG and the overall population. Higher proportions of participants treated with  rozanolixizumab versus placebo achieved a 2 point or greater improvement in MG-ADL and a 3  point or more improvement in Quantitative Myasthenia Gravis (QMG) scores and Myasthenia Gravis Composite (MGC) scores. Adverse effects associated with treatment included headache, diarrhea, pyrexia, and nausea.

“People living with MG 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. We are firmly committed to supporting the gMG community by providing solutions to help improve outcomes for patients and reduce the day-to-day burden of the disease,” said Charl van Zyl, executive vice president Neurology Solutions & Head of EU/International Markets, UCB. 

With a FDA priority review, a decision regarding the application is expected within approximately 6 months compared with non-priority reviews. According to details provided by the FDA, a priority review is granted to medicines “that could deliver significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions if approved when compared to standard applications.”