Santhera: UK marketing authorization application for Duchenne muscular dystrophy treatment

In parallel to the MAA submission to the UK MHRA, Santhera is currently preparing an application to include vamorolone for the treatment of DMD in the Early Access to Medicines Scheme (EAMS) in the UK. The aim of the EAMS is to provide patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization, when there is a clear unmet medical need.

“We are proud to have completed the third submission for vamorolone in DMD to a major regulatory agency within a few months. This underpins our determination to bring a new treatment to patients in need of effective and well-tolerated therapies,” said Shabir Hasham, MD, CMO of Santhera. “We look forward to working closely with the MHRA during the MAA and EAMS review process with the goal of quickly providing an emerging therapy to patients in the UK.”

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 efficacy and safety of vamorolone (2 and 6 mg/kg/day) versus placebo and prednisone (0.75 mg/kg/day), 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].

In the U.S., the Food and Drug Administration (FDA) has set October 26, 2023, as the Prescription Drug User Fee Act (PDUFA) target action date upon which approval of the new drug application (NDA) for vamorolone in DMD is expected. In the EU, a corresponding MAA has been validated and is under review by the European Medicines Agency (EMA) with an expected approval in late 2023. Subject to approvals, Santhera plans to launch vamorolone in both the U.S. and the EU in Q4-2023.

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.

References
[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