PPMD was recently joined by Catalyst Pharmaceuticals to review the recent FDA approval of AGAMREE (vamorolone), a new steroid approved for the treatment of Duchenne. 

Amy Grover, Catalyst’s Senior Director of Patient Advocacy, introduced the Catalyst team, including Alison Anderson, Medical Affairs Director. Alison provided an overview of AGAMREE, which is approved for individuals aged two years and older and comes as an orange-flavored oral liquid dosed as 6mg/kg once daily by mouth. She also reviewed common side effects, precautions, and clinical trial data that supported the FDA’s approval of the medication. You can learn more about the clinical trial data behind AGAMREE from our recent approval webinar with Dr. Aravindhan Veerapandiyan here. 

Maria Pandolfo, Sr. Vice President of Patient Services then provided an overview of Catalyst Pathways, Catalyst’s patient support program. The Catalyst Pathways team consists of several care coordinators, insurance navigators, and patient access liaisons who work together to facilitate access to medication, navigate insurance approval, ensure bridge supply when needed, and answer questions throughout the access process. While families can sign up for Catalyst Pathways on yourcatalystpathways.com beginning on December 1, 2023, access to AGAMREE is expected in the first quarter of 2024.

The Catalyst team then answered community questions. Some highlights include:

• There are plans for a tablet formulation of AGAMREE, but no timeline is available
• AGAMREE must remain refrigerated once the bottle has been opened and is good for 90 days
• Long-term data about fractures, the heart, and lungs is not yet available, but will be shared publicly in the future as more data becomes available
• AnovoRx is the speciality pharmacy who will distribute AGAMREE 
• While there are only English-speaking staff on the Catalyst Pathways team at the time of this writing, there is a translation service available for non-English speakers. 
• The recent approval of AGAMREE is specific to the United States; rollout timelines outside of the US remain unclear, and commercial access to AGAMREE in Europe will be handled by Santhera. 
• The price of AGAMREE has not been shared, but is anticipate to be “at a slight discount to Emflaza”

We look forward to learning more about the commercialization of AGAMREE in the coming weeks, and are eager to partner with you in answering your questions about care, treatment options, and more. If you have more questions, email the PPMD Care Team at careteam@parentprojectmd.org, schedule a PPMD For You appointment, and stay up to date at parentprojectmd.org. 

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