Defeat Duchenne Canada is pleased to share a recent update on vamorolone and the next steps in the approval process.
Catalyst Pharmaceuticals announced the completion of its acquisition from Santhera Pharmaceuticals Holdings (“Santhera”) of an exclusive license for vamorolone in North America, a potential dissociative anti-inflammatory steroid treatment for people with Duchenne muscular dystrophy. The license is for exclusive commercial rights in the U.S., Canada, and Mexico.
In clinical studies, vamorolone demonstrated efficacy with a significant reduction of steroid-associated side effects and benefits for bone health, growth, and behaviour, offering the potential to address an important unmet medical need in DMD patients. Vamorolone has received FDA Orphan Drug and Fast Track designations and has been granted a PDUFA (Prescription Drug User Fee Act) action date of October 26, 2023.
“We believe that vamorolone, if approved, has the potential to deliver significant near and long-term value and to be a very meaningful advancement to the current standard of care paradigm for DMD patients.
“Vamorolone fortifies our neuromuscular portfolio with an innovative therapy that, in clinical studies, demonstrated an enhanced safety and tolerability profile as compared to prednisone. We plan to include vamorolone for DMD patients in our Catalyst Pathways® specialty pharmacy program to help ensure that all patients have access to the full patient benefits of the program. Our planned strategy to facilitate access to vamorolone underscores our steadfast commitment to improving the lives of patients suffering from rare neurological conditions.”Patrick J. McEnany, Chairman and CEO of Catalyst
Defeat Duchenne Canada will continue to monitor and provide updates on the progress of the vamorolone application to Health Canada.