FDA Approves Steroid-Like Drug for Duchenne Muscular Dystrophy

WASHINGTON — Boys with Duchenne muscular dystrophy (DMD) have a clinically proven, new treatment option: The U.S. Food and Drug Administration’s recently approved vamorolone, a steroid-like, anti-inflammatory drug developed based on research performed at the Children’s National Hospital, Washington DC.DMD Drug  

Created by ReveraGen BioPharma Inc., vamorolone has a molecular structure similar to traditional corticosteroids that are currently used to treat DMD. Yet its structure was found to be chemically different enough to reduce unwanted side effects, including brittle bones and reduced stature. 

Nearly two decades ago, ReveraGen leaders—president and CEO Eric Hoffman, PhD, and vice president for research Kanneboyina Nagaraju, DVM, PhD—launched research efforts into the drug when they led the Center for Genetic Medicine Research at Children’s National. They worked with then-chief academic officer Mark Batshaw, MD, on the new clinical option. “Throughout my career, I have treated children with DMD, and I have seen over time how their shorter heights and brittle bones impact them physically and emotionally—in terms of their self-esteem and ability to participate in activities,” Batshaw said. “This drug should help these boys function more effectively and prevent certain long-term complications.”

How is vamorolone better than other Duchenne muscular dystrophy treatments?  

Muscular dystrophy includes a group of degenerative genetically inherited neuromuscular diseases that affect only boys. DMD is the most common, severe, and life-threatening form of muscular dystrophy. 

ReveraGen studied vamorolone in patients aged two years and up in the hopes of providing a new, FDA-approved treatment option for affected children. In clinical trials, daily treatment with vamorolone improved muscle strength and stature with results comparable to prednisolone, but without some of the most impactful side effects of steroids, particularly the stunted growth and weakened bones. 

“We cannot wait to see the tremendous effort behind vamorolone in the hands of patients and clinicians treating Duchenne muscular dystrophy,” said Kolaleh Eskandanian, PhD, MBA, PMP, vice president and chief innovation officer for Children’s National. “Today’s FDA approval for ReveraGen shows the importance of supporting clinicians and researchers who are developing solutions to advance health care for children.”  

Hoffman said the drug has been through a series of clinical trials showing advantages over the current treatment options. In 2024, Catalyst Pharma will market vamorolone under the trade name Agamree in the United States.

- This press release was originally published on the Children's National Hospital website