• Monday, October 31, 2022 @ 12:00 am

Santhera Pharmaceuticals (SIX: SANN) announces that the European Medicines Agency (EMA) has validated its marketing authorization application (MAA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD). Validation confirms that the submission is complete and that the review by the EMA’s Committee for Medicinal Products for Human Use (CHMP) has begun.

“The initiation of the CHMP review of our application is an important milestone for patients with DMD and a significant acknowledgement for our Company. We are looking forward to working closely with the rapporteurs and CHMP during the review process to make vamorolone available to patients as soon as possible,” said Shabir Hasham, MD, CMO of Santhera.

Santhera expects the CHMP to complete the review and issue an opinion regarding approval to EMA’s European Commission (EC) in late Q3-2023. Subject to EC approval, expected later in 2023, vamorolone will receive marketing authorization in all member states of the European Union, as well as in Norway, Liechtenstein and Iceland.

In the U.S., Santhera and ReveraGen have just completed the submission of the new drug application (NDA) to the Food and Drug Administration (FDA), seeking priority review for vamorolone for the treatment of DMD. Subject to approval, this paves the way for a potential launch in H2-2023.

Vamorolone has been granted Orphan Drug status in the U.S. and in Europe for DMD, and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD.

[1]   Guglieri M et al (2022). JAMA Neurol. Published online August 29, 2022. doi:10.1001/jamaneurol.2022.2480. Link.
[2]   Mah JK et al (2022). JAMA Netw Open. 2022;5(1):e2144178. doi:10.1001/jamanetworkopen.2021.44178. Link.
[3]   Guglieri M et al (2022) JAMA. doi:10.1001/jama.2022.4315
[4]   Heier CR et al (2019). Life Science Alliance DOI: 10.26508
[5]   Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293

You may also be interested in