UCB announced that the FDA accepted the company's filing to review a Biologic License Application (BLA) for its investigational treatment rozanolixizumab, and that the Agency has granted Priority Review. Rozanolixizumab is a subcutaneous (SC) monoclonal antibody targeting the neonatal Fc receptor (FcRn) for the treatment of adults with generalized myasthenia gravis (gMG) who are anti-acetycholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
A FDA Priority Review designation is typically granted by the Agency to a medicine which, if approved, could deliver significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.3 Priority Review designation means the FDA's goal is to take action on an application within 6 months, compared to 10 months under standard review. In 2019, the U.S. FDA granted orphan drug designation to rozanolixizumab for the treatment of MG.
The safety and efficacy of rozanolixizumab have not been established and they are not currently approved for use in any indication by any regulatory authority worldwide.
The FDA Priority Review designation follows the recent European Medicines Agency (EMA) validation of the Marketing Authorization Application (MAA) for rozanolixizumab for the treatment of adults with AChR or MuSK antibody positive gMG who require treatment in addition to steroids or non-steroidal immunosuppressants. Validation confirms that the application is complete and the formal review process by the EMA's Committee for Medicinal Products for Human Use (CHMP) can begin. Orphan designation was granted by the European Commission in April 2020 to rozanolixizumab for the treatment of myasthenia gravis.
UCB expects to receive feedback from both the FDA and EMA during the second quarter of 2023.
"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. "The FDA's decision to assess rozanolixizumab via their priority review process, as well as the recent filing of the MAA in Europe, brings us important steps further on our journey towards approvals for rozanolixizumab. We look forward to working with the FDA and EMA to help bring this new treatment option to patients."
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox!
Sign up now!