Exondys 51: First Drug to Treat DMD Gets Approval from US FDA

The US regulatory authority on 18th Sep approved Exondys 51 (eteplirsen), Sarepta Therapeutics’ initial drug to treat individuals with Duchenne muscular dystrophy (DMD), an unusual genetic problem that leads to progressive muscle deterioration and weakness in young kids.

The acceptance is extremely controversial following a FDA advisory committee voting against acceptance in April this year as the external experts stated there was not significant proof that the drug is effective in offering clinical advantage, which is the standard for approval. Prior to that vote and afterwards, the DMD patient community protested strongly.

DMD, taking place in around 1 in every 3,600 male infants globally, is triggered by lack of dystrophin, a protein that assists keep muscle cells intact. The initial symptoms are generally seen between the ages of 3 and 5, and get worse eventually.

US FDA stated that following the hearing, Sarepta submitted further data “demonstrating significant proof of dystrophin production, even though the quantity of dystrophin produced was only a small fraction of the normal level.”

Exondys 51, is particularly indicated for sufferers who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping Drug, who represent roughly 13% of the population with DMD. After getting approval from US FDA, Sarepta said that the average cost per individual will be $300,000.

Exondys 51, was accepted under FDA’s accelerated approval program, reserved for medication to treat severe or life-threatening conditions, and where there is a absence of accessible treatment.

“Depending on the data presented by the Sarepta Therapeutics, US FDA has came to the conclusion that there is a statistically considerable raise in dystrophin production in indicated sufferers who are subjected to the drug that meets this requirement,” FDA said.