On October 6, 2022, Santhera Pharmaceuticals provided an update regarding their submission of an authorization application to the European Medicines Agency for vamorolone in Duchenne muscular dystrophy. A press release regarding the initial submission of application can be found here.
Please find their EMA authorization application update below.
Dear Duchenne community,
We are writing to provide a few more details about our recent filing of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vamorolone for the treatment of Duchenne muscular dystrophy.
As you may have read in a Santhera press release on Oct. 3, 2022, the MAA is based on positive data from several studies, including the Phase 2b VISION-DMD study, open-label studies, and an external comparator study. We have attached the press release here for your reference about vamorolone, the VISION-DMD study, and additional details about the EMA filing.
Vamorolone has been granted Orphan Drug status in the European Union. Subject to successful validation of the MAA, a decision from the EMA is expected during the second half of 2023. In the U.K., the Medicines and Healthcare Products Regulatory Agency (MHRA) has designated vamorolone with Promising Innovative Medicine status. We will keep the community updated about our submission for vamorolone to the MHRA.
The U.S. Food and Drug Administration (FDA) has also granted vamorolone Orphan Drug status, as well as Fast Track and Rare Pediatric Disease designations. As stated in Santhera’s press release on June 29, 2022, completion of the FDA filing was postponed 4-6 months to Q4 2022 after a third-party contract manufacturing organization communicated a delay in establishing FDA-inspection readiness. The filing of the new drug application (NDA) to the FDA for vamorolone is expected to be complete by the end of 2022.
In closing, we would like to take this moment to express our deep appreciation to the global Duchenne community for its support of the vamorolone development program as undertaken by Santhera and its partner ReveraGen. As a company, Santhera is unyielding in our support of research to support the Duchenne community. On behalf of Santhera, we will continue to keep you posted on our progress.
If you have any questions or we can be of any further support, please do not hesitate to contact Santhera’s Patient Advocacy Consultant Mindy Cameron at email@example.com.