Santhera Pharmaceuticals files VBP 15 for Duchenne muscular dystrophy with UK MHRA and applying to EAMS
Santhera Pharmaceuticals announced that it has submitted a marketing authorization application (MAA) to the UK Medicines and Healthcare products Regulatory Agency (MHRA) for VBP 15 (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD)
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.
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