MAA submitted to the UK MHRA for vamorolone in Duchenne muscular dystrophy
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.
At the core of the MAA submission are positive data from the pivotal Phase IIb 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 . 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.
In the U.S., the 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.
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