October 26, 2023 / Care,Clinical Trials,Research

FDA Grants Approval to AGAMREE® (Vamorolone)

PPMD is excited to learn that the FDA has granted approval to AGAMREE® (vamorolone), a dissociative steroid indicated for individuals diagnosed with Duchenne muscular dystrophy from age 2 years and older. 

AGAMREE® is a first-in-class drug that works similarly to corticosteroids (anti-inflammatory medications used to help maintain muscle strength). AGAMREE® aims to retain the beneficial anti-inflammatory and muscle-strengthening aspects of corticosteroids, while decreasing some of the undesirable side effects (bone fragility, stunted growth, insulin resistance, mood changes, delay of puberty and others).

To find out if you or your child is eligible to receive AGAMREE® and if your neuromuscular center is able to prescribe it, call your neuromuscular team, who will be able to provide you with the most appropriate information on next steps. If you have nonmedical questions about AGAMREE®, you can visit yourcatalystpathways.com or call 1-833-422-8259.

PPMD will also begin including AGAMREE® on our Access & Coverage Resources page in the coming weeks as more information becomes available. We remain committed to working towards community access to all approved products and will continue to work with all relevant stakeholders to ensure a favorable access environment for our community.

What Does This Approval Mean?

Nov 1 Webinar: What this means for you and your family

PPMD will host a community webinar on Wednesday, November 1st at 1:00 PM ET with Dr. Aravindhan Veerapandiyan (Arkansas Children’s Hospital) to discuss the approval of AGAMREE® and what it means for individuals living with Duchenne. Registration is open for the webinar and attendees can submit questions in advance here.

Register >

Nov 15 Webinar: PPMD & Catalyst Pharmaceuticals

PPMD will also host a webinar on Wednesday, November 15th at 1:00 PM ET with Catalyst Pharmaceuticals, the drug company responsible for the commercialization of AGAMREE®, to provide an explanation of the drug, its label, and eligibility criteria. Register now and submit your questions in advance here.

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Additionally, the PPMD team has compiled a resource of FAQs surrounding the approval of AGAMREE® and what this means, including questions on eligibility and access.

View FAQ >

Community Impact

For nearly three decades, PPMD has been dedicated to working with researchers, clinicians, industry, and the Duchenne community to advance treatments for all people living with Duchenne. PPMD is pleased to have collaborated with other foundations and community partners to support the early development of VPB15 (vamorolone). 

We extend our heartfelt gratitude to the patients and their families who participated in these trials, as well as to ReveraGen BioPharma, Santhera Pharmaceuticals, and the many dedicated clinicians and scientists who have tirelessly worked to develop this therapy. 

We are thrilled to see the approval of another therapeutic option for Duchenne. As we strive to ensure that these approved therapies become accessible to all who need them, today, we unite to celebrate another remarkable milestone.

PPMD’s Funding

PPMD provided philanthropic investment to ReveraGen for the development of VPB15 (vamorolone). As part of that agreement PPMD will recover that investment and additional payment from ReveraGen if vamorolone attains specific commercial milestones. Any returns will be reinvested in programs supporting PPMD’s mission. PPMD has a comprehensive approach to identify opportunities to accelerate development of all therapies in Duchenne, including regulatory, research, and patient-recruitment counsel to help expedite the progress of investigational products.

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