The United States Food and Drug Administration has recently approved Eteplirsen from Sarepta Therapeutics of Cambridge, Massachusetts for treating patients with Duchenne Muscular Dystrophy(DMD).
Muscular Dystrophy is a rare genetic disorder characterized by muscle deterioriation and weakness. This disease occurs due to the absence of dystrophin protein which is essential for keeping the muscle cells intact. It usually affects boys between the age of 3 and 5.About 9000 -12000 boys in the United States were affected with Duchenne Muscular Dystrophy. These boys usually need a wheel chair during their early 20s.
‘Eteplirsen receives accelerated approval from FDA for the treatment of Duchenne Muscular Dystrophy.’
Eteplirsen (Exondys 51) is mainly given for patients with mutation in dystrophin gene. The drug has received accelerated approval from FDA for treating life threatening conditions. Clinical trials were required to confirm the benefits of the drug. Around 12 boys with Duchenne Muscular Dystrophy were tested using eteplirsen drug. Common side effects like balance disorder and vomiting were reported by the participants.
The drug acts by a technology known as the Exon skipping, by which the functional form of dystrophin is produced. And was found to be applicable only for 13% of the patients with duchenne muscular dystrophy.
Dr. Janet Woodcock, Director of the F.D.A.'s Center for Drug Evaluation and Research,said that "We eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval."
The agency has also asked Sarepta therapeutics to compare the efficacy of the drug with placebo control. And if the drug fails to answer then the agency must initiate proceedings for withdrawing the approval.