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Santhera: Marketing Authorization Application to the European Medicines Agency for Vamorolone

October 4, 2022

Santhera Pharmaceuticals has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).

At the core of the MAA submission are positive data from the pivotal Phase 2b VISION-DMD study, which comprised a (1) 24-week period to demonstrate the efficacy and safety of vamorolone (2 and 6 mg/kg/day) versus prednisone (0.75 mg/kg/day) and placebo, followed by a (2) 24-week period to evaluate the maintenance of efficacy and collect additional longer-term safety and tolerability data [1]. In addition, the filing includes data from three open-label studies in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months [2].

“Duchenne is a debilitating disease with patients in need for effective and well-tolerated therapies. Our EU submission marks an important step towards making vamorolone available to DMD patients in the respective European markets and we look forward to working closely with the EMA during the review process.”

Shabir Hasham, MD, CMO of Santhera

In the U.S., Santhera expects to complete the filing of the new drug application (NDA) for vamorolone to the Food and Drug Administration (FDA) in Q4-2022.

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.
[2] Mah JK et al (2022). JAMA Netw Open. 2022;5(1):e2144178. doi:10.1001/jamanetworkopen.2021.44178.

Defeat Duchenne Canada continues to work with Santhera for application to Canada. Join us at our Family Forum on November 4-6, 2022, where Santhera Pharmaceuticals will provide additional updates and an opportunity for Q&A.

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