Groundbreaking Duchenne treatment approved by the FDA

July 20 • 2023

The Food and Drug Administration (FDA) has given the green light to a new gene therapy treatment for Duchenne Muscular Dystrophy (DMD) patients 4-to-5-years-old. The life-extending therapy, ELEVIDYS (delandistrogene moxeparvovec-rokl), was discovered through Muscular Dystrophy Association (MDA) research funded by donors like the IAFF. 

“Because we have partners like the IAFF that believe in and generously support our mission, MDA has been able to fund critical research, facilitate innovative clinical trials, and gather the necessary resources to accelerate the development of this revolutionary treatment,” said Donald Wood, Ph.D., President and CEO of MDA. “The approval of ELEVIDYS signifies a major leap forward in our collective efforts to combat Duchenne Muscular Dystrophy and provides hope to existing patients and their families.” 

“The IAFF has been proud to be MDA’s steadfast partner for nearly 70 years,” said General President Edward Kelly. “We go out and fill the boot and conduct a host of other fundraisers every year for MDA because we know every dollar we donate helps develop life-improving therapies like ELEVIDYS. This latest FDA approval is very exciting news, and we know even more are on the horizon.” 

DMD is the most common form of muscular dystrophy in pediatric patients and mostly occurs in males. It is caused by a genetic variant in the dystrophin gene that reduces the amount of dystrophin protein in muscles, which increasingly inhibits their ability to function. Over time, DMD becomes fatal as muscle degeneration disrupts lung and heart function. 

Most recent treatments include drugs like Exondys 51 and Vyondys 53, which use a technology called “exon-skipping” that allows the body to produce more of the dystrophin protein. This strategy does not cure DMD but slows down its progression. 

ELEVIDYS is a different type of medical technology known as gene replacement therapy. The treatment calls for injecting a shortened version of the dystrophin gene using the adeno-associated virus (AAV) (no symptoms) as the carrier.  

“This is an exciting time as more gene therapies for neuromuscular diseases enter clinical translation. The approval of ELEVIDYS is ground-breaking as it represents the first gene therapy for DMD and is designed to target the underlying cause of the disease,” said Sharon Hesterlee, Ph.D., MDA Chief Research Officer. “What is truly great about this new therapy is that there only needs to be one infusion instead of the multiple infusions that were needed with previous therapies.”

FDA approved the therapy for 4 and 5-year-olds as research has shown marked improvement for that age group. As ongoing research continues to generate data, MDA remains optimistic ELEVIDYS will be approved for additional age groups in the coming months.