MAA submitted to EMA for vamorolone for the treatment of Duchenne muscular dystrophy
Santhera Pharmaceuticals announces that the Company 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 IIb VISION-DMD study which comprised a 24-week period to demonstrate efficacy and safety of vamorolone (2 and 6 mg/kg/day) versus prednisone (0.75 mg/kg/day) and placebo, followed by a 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., Santhera expects to complete the filing of the new drug application (NDA) for vamorolone in DMD to the FDA in Q4-2022.
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