FDA Sets Fall Date for Approval Decision on Vamorolone for DMD
Corticosteroid treatment also under review by regulatory agencies in Europe
The U.S. Food and Drug Administration (FDA) is reviewing vamorolone for Duchenne muscular dystrophy (DMD) and has set a target action date of Oct. 26 for its decision on whether to approve the dissociative corticosteroid treatment.
The regulatory agency accepted a new drug application (NDA) for the experimental therapy — news that was welcomed by developers Santhera Pharmaceuticals and ReveraGen BioPharma — and said it is not planning to hold an advisory committee meeting.
Santhera already had submitted a marketing authorization application to the European Medicines Agency for the therapy’s approval in the EU. That application also was accepted and is now under review.
“We are delighted that the FDA has accepted Santhera’s vamorolone NDA for filing. We believe this product addresses a clear unmet medical need,” Dario Eklund, CEO of Santhera, said in a joint press release.
Added Eric Hoffman, PhD, president and CEO of ReveraGen: “If approved, vamorolone will emerge as an additional treatment option in 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.”
Companies seeking regulatory approval in US, Europe
Regulatory agencies in both Europe and the U.S. have taken steps to help speed the development and regulatory review of vamorolone as a therapy for DMD.
It received orphan drug status in the U.S. and Europe, as well as fast track and rare pediatric disease designations from the FDA. The therapy also was awarded promising innovative medicine status in the U.K.
Treatment guidelines for DMD, the most common type of muscular dystrophy, recommend that patients be treated with corticosteroids — anti-inflammatory medications that can help preserve muscle tissue and delay muscle weakness.
However, corticosteroid use can have several side effects, including fluid retention and weight gain, mood changes, and high blood pressure.
Vamorolone, a first-in-class dissociative corticosteroid, was designed to have the anti-inflammatory activity of corticosteroids, but with fewer side effects. It is administered orally as a flavored liquid.
The submissions for approval of vamorolone were supported mainly by results from the Phase 2b VISION-DMD trial (NCT03439670). The trial involved 121 boys with DMD, ages 4-6 and able to walk. Each was randomly assigned to receive vamorolone daily at 2 or 6 mg/kg, the corticosteroid prednisone at 0.75 mg/kg, or a placebo.
After the first 24 weeks (about six months) of treatment, boys treated with vamorolone had significant improvements compared with those on the placebo in several motor function assessments. These included the time to stand test, which assesses the speed at which a person stands from a lying position, and the six-minute walk test, a measure of the distance a person can walk in six minutes.
In the trial extension, in which all participants received vamorolone for a further 24 weeks, the treatment continued to show sustained improvements.
In both parts of the trial, vamorolone treatment showed a good safety and tolerability profile at both doses. Side effects of vamorolone were generally mild to moderate in severity, and the treatment was associated with fewer side effects than prednisone.
“The data generated across the clinical trial program indicate that vamorolone has the potential to address relevant aspects in patient care that could also enhance treatment outcomes,” Hoffman said.
Eklund added that the companies “look forward to working closely with U.S. regulators to advance vamorolone towards approval.”
If the FDA’s decision in October is positive, and vamorolone is approved, Santhera is planning to launch the new DMD therapy in the U.S. in the last quarter of this year.