The anti-inflammatory treatment is for Duchenne muscular dystrophy
Santhera has announced that the company has submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for vamorolone. The therapy is used for the treatment of Duchenne muscular dystrophy (DMD).
At the core of the MAA submission is positive data from the pivotal phase 2b Vision-DMD study that comprised a 24-week period to demonstrate efficacy and safety of the drug versus prednisone (0.75mg/kg/day) and placebo.
This was 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 as two different doses for a total treatment period of up to 30 months.
Vamorolone is an investigational anti-inflammatory drug candidate with a mode of action based on binding to the same receptor as corticosteroids.
“Duchenne is a debilitating disease, with patients in need of effective and well-tolerated therapies,” explained Shabir Hasham, chief medical officer of Santhera. “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.”
In the US, Santhera expects to complete the filing of the new drug application for vamorolone with the Food and Drug Administration (FDA) by the end of 2022.
Vamorolone has already been granted Orphan Drug status in the US and Europe for DMD, and has received Fast Track and Rare Paediatric Disease designations from the US FDA and Promising Innovative Medicine (PIM) status from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for DMD.