The FDA just recently placed investigational measures on Sarepta Therapeutics new medication “Elevidys” that has been indicated for treatment patients suffering from Duchenne muscular dystrophy (DMD), after alarming 3 death related cases potentially after administration of medication.
Preliminary reports indicate that the three fatalities were linked to acute liver failure in patients who had received Elevidys or an investigational gene therapy utilizing the same AAVrh74 serotype as Elevidys. One of the deaths occurred in a clinical trial conducted under an investigational new drug application for the treatment of Limb-Girdle Muscular Dystrophy.
Elevidys is an adeno-associated virus vector-based gene therapy It works by delivering a gene that enables the body to produce Elevidys micro-dystrophin a shorter version of the normal dystrophin protein to help muscle function. The treatment is given as a single intravenous dose.
Duchenne muscular dystrophy is a rare genetic disease occurs due to a faulty gene that results in irregularities in, or absence of, dystrophin, a protein that helps keep the body’s muscle cells intact.
Elevidys was fully approved on June 20, 2024, for ambulatory DMD patients aged 4 and older with a confirmed DMD gene mutation. It had previously received accelerated approval on June 22, 2023, for non-ambulatory patients, with continued approval dependent on confirmatory trials.
Following new safety concerns, the FDA has restricted its use to ambulatory patients and is investigating the risk of acute liver failure, including serious outcomes such as hospitalization and death, linked to Sarepta’s AAVrh74 platform. Further regulatory actions may follow.
