PPMD Applauds FDA for Approval of AGAMREE® (Vamorolone) for Duchenne Patients Ages Two Years and Older

News provided by

Parent Project Muscular Dystrophy (PPMD)

Oct 26, 2023, 19:11 ET

WASHINGTON, Oct. 26, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), welcomed today's decision by the Food and Drug Administration (FDA) to approve AGAMREE® (vamorolone) a dissociative steroid indicated for individuals diagnosed with Duchenne from age two years and older. AGAMREE® marks a significant milestone as the second therapy approved for Duchenne that is applicable regardless of the specific genetic mutation. It is the seventh approved therapy for Duchenne in the United States. AGAMREE® will be made available and marketed in the United States by Catalyst Pharmaceuticals, which entered into an agreement with Santhera Pharmaceuticals in June 2023 for exclusive North America commercialization rights.

"PPMD is thrilled that the FDA has approved AGAMREE®, a steroid alternative therapy that aims to retain the beneficial anti-inflammatory and muscle strengthening aspects of corticosteroids, while decreasing some of the undesirable side effects such as bone fragility, delayed growth, insulin resistance, and behavior changes. Today's approval has the potential to offer patients a therapy option with better tolerability, addressing a significant unmet need in our community," said PPMD Founding President and CEO Pat Furlong.

"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 also want to commend FDA leadership for recognizing the potential of AGAMREE® to make a difference in the lives of individuals living with Duchenne," Furlong added. "PPMD is pleased to have collaborated with other foundations and community partners to support the early development of vamorolone. We are thrilled to see the approval of another therapeutic option for Duchenne."

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. Register now and submit your questions in advance.

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.

"Going forward, PPMD remains committed to working with all stakeholders and the community to do all we possibly can to ensure all patients who are eligible for AGAMREE® obtain access to the drug. It is not enough to clear the FDA. We must make sure that all those who can benefit from these innovative therapies are able to access them as they reach the market," said Furlong. "Additionally, PPMD believes that it will take a combination of therapies to fully halt progression and will continue to support the development of additional tools and resources to accelerate the development and approval of additional therapies and treatments for Duchenne and Becker muscular dystrophy."

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 employs 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. PPMD's patient-report registry, The Duchenne Registry, as well as PPMD's Duchenne Outcomes Research Interchange, serve as resources for researchers and industry to access aggregated, de-identified information provided by patients and families. Additionally, PPMD remains committed to its sector-leading patient engagement efforts, which include multiple patient preference studies and a revised community-generated draft guidance document, recently updated to reflect advancements in knowledge, understanding, care, clinical trials, and approvals. PPMD also takes a lead role in advocating for national newborn screening efforts for Duchenne, Becker, and carrier identification, as well as policy efforts around access and reimbursement.

ABOUT PARENT PROJECT MUSCULAR DYSTROPHY

Duchenne is a genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won seven FDA approvals.

Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.

SOURCE Parent Project Muscular Dystrophy (PPMD)