The FDA’s Ruling on Vamorolone in DMD

You may have heard about the FDA’s ruling, which approved vamorolone as an alternative steroid for use in Duchenne muscular dystrophy.  

Vamorolone is a new steroid alternative with fewer side effects than prednisone. The drug aims to preserve the beneficial anti-inflammatory and muscle-strengthening characteristics of corticosteroids, while decreasing some of the unwelcome side effects, including bone fragility, delayed growth, and behavior changes.

As we learned in this announcement, vamorolone was found to be both safe and effective as a therapy for Duchenne muscular dystrophy in its newly approved indication.

“We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with DMD and potentially other chronic inflammatory diseases,” said Patrick McEnany, chairman and CEO of Catalyst Pharmaceuticals.” Catalyst Pharmaceuticals recently acquired North American distribution rights for vamorolone.

Vamorolone will need more study in juvenile myositis before most pediatric rheumatologists will be comfortable prescribing it for juvenile myositis.

“We remain urgently focused on working with the FDA and with the vamorolone developers to conduct a safety and efficacy trial in juvenile myositis. We are so grateful to Drs. Eric Hoffman and Kanneboyina Nagaraju of the Cure JM Medical Advisory Board for their dedication and fundamental research that led to the creation of vamorolone. Dr. Hoffman presented how vamorolone acts and why it is superior to prednisone in a September Town Hall presentation to Cure JM families,” said Andrew Heaton, Ph.D., Chief Scientific Officer, Cure JM Foundation.

Drs. Eric Hoffman and Kanneboyina Nagaraju have led fundamental research during vamorolone’s development.

Cure JM Foundation’s plan for a vamorolone clinical trial in juvenile myositis is now before the FDA. We are hopeful that this approval will help clear the path for accelerated testing of the drug in juvenile myositis. We are particularly grateful to the team at Cure JM’s Center of Excellence at Duke University for taking the lead in planning and serving as the principal site for a future trial of vamorolone in JM.  

Although there is still work ahead, there is optimism that this news is a significant step in the right direction as we continue fulfilling our mission of finding better treatments for all JM patients.  

As we contemplate this extraordinary news, we are particularly grateful to you. Your support allowed Cure JM to provide crucial early funding to the developers of vamorolone. Vamorolone is just one of the new treatments that our esteemed researchers are investigating, some of which I shared with you here earlier this month.

We are grateful to have you by our side as we move this critical work forward for our children, and look forward to reporting back to you on our progress.

Rare Disease Warriors

Since it is Rare Disease Month, we would like to take a moment and shine the spotlight on some of our amazing JM warriors.

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