Department of Defense
United States Army Medical Research and Development Command
Congressionally Directed Medical Research Programs
Released: November 3, 2023
CDMRP-Funded Research Contributed to a Recently FDA-Approved Drug for Duchenne Muscular Dystrophy
Vamorolone offers individuals with Duchenne muscular dystrophy a new treatment option, improving quality of life and preventing long-term complications
FORT DETRICK, Md. – In October 2023, the FDA approved a new drug, vamorolone, for the treatment of Duchenne muscular dystrophy, offering individuals with this disease a new option to improve quality of life and preventing long-term complications. CDMRP-funded research contributed to the early development of this treatment option now approved for patients two years old and older.
“CDMRP funds research to accelerate the transformation of therapeutic ideas into useful clinical applications,” Col. Sarah Goldman, director of CDMRP, said. “Our organization is extremely proud to learn vamorolone is now FDA-approved and will be accessible to those who need the treatment.”
Eric Hoffman, Ph.D., professor of pharmaceutical sciences at Binghamton University and president and CEO of ReveraGen BioPharma, Inc., the company responsible for developing vamorolone, led early research efforts contributing to development of vamorolone with support from a series of Institutionally Based Research Program awards to Children's National Medical Center managed by CDMRP.
As part of these research awards, Hoffman sought to better understand disease severity and responses to therapy in models of Duchenne muscular dystrophy. Further studies in patient populations demonstrated treatment with vamorolone improved muscle strength comparable to prednisone, a commonly used corticosteroid.
Vamorolone demonstrated an improved safety and tolerability profile than current standard-of-care corticosteroids and no association with the side effects of stunted growth, weakened bones, and mood disturbances, improving the quality of life for those affected by DMD.
According to Hoffman, vamorolone is the first dissociative steroidal anti-inflammatory that retains efficacy with no stunting of growth or no harmful changes in bone biomarkers for those with Duchenne muscular dystrophy.
“The CDMRP provided seminal research funding to my lab at Children's National Medical Center in 2005 that germinated the idea of development of a dissociative steroidal anti-inflammatory drug,” Hoffman said. “The focus of the CDMRP program on translational research enabled us to carry out lead compound selection, moving vamorolone forward through early drug development, and leading to the spin-off of ReveraGen in 2008."
According to recent research, Duchenne muscular dystrophy is an inherited form of muscular dystrophy that is currently fatal and is characterized by a severe loss of function. An X-linked disease, a majority of diagnoses affect boys. Symptoms typically appear between the ages of two and three years old. By 12 years old, many patients are confined to wheelchairs. In the disease, the protein dystrophin is missing from all muscles, such as the heart and smooth muscles of the gut and vascular systems. Alternatively, muscles are fibrous connective tissues and fat.
This drug’s approval is based on decades of international collaborative research and development.
For more information about CDMRP and our research programs, please visit our website at https://cdmrp.health.mil.
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Last updated Friday, November 3, 2023