Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc announce that they have completed the rolling submission of a new drug application (NDA) to the U.S. Food and Drug Administration (FDA), seeking priority review for vamorolone for the treatment of Duchenne muscular dystrophy (DMD). Subject to approval, vamorolone is set to become available to patients in the U.S. in H2-2023.
At the core of the NDA submission are positive data from the pivotal Phase 2b VISION-DMD study which comprised (1) 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 (2) a 24-week period to evaluate the maintenance of efficacy and collect additional longer-term safety and tolerability data . In addition, the filings include data from four open-label studies (including extension) in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months  and an external comparator study.
“Completion of the vamorolone NDA submission is a major step towards our goal of bringing this investigational therapy to patients living with DMD, and represents a vital milestone for Santhera,” said Dario Eklund, CEO of Santhera. “We look forward to working closely with U.S. regulators to advance vamorolone towards approval.”
“We would like to express our deepest gratitude to the study participants, their families and caregivers, as well as the investigators and study staff. Their unwavering commitment enabled us to achieve this milestone,” said Eric Hoffman, PhD, President and CEO of ReveraGen BioPharma. “If approved, vamorolone will emerge as an addition to current standards of care in DMD with the potential to address unmet medical needs and treat a majority of Duchenne patients starting at an early age.”
With the completion of the rolling NDA submission, Santhera and ReveraGen have also applied for priority review. Typically, within 60 days of the receipt of the dossier, the FDA will inform if a priority review will be granted. A priority review designation indicates FDA’s goal is to take action on an application within six months (compared to ten months under standard review) which would set an anticipated approval date for as early as mid-2023.
In Europe, Santhera has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) in September 2022, seeking approval for vamorolone in the European Union, as well as in Norway, Liechtenstein and Iceland.
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.
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