The underlying cause of Duchenne muscular dystrophy is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function. In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. The nod was based on the surrogate endpoint of an increase in dystrophin in skeletal muscle observed in some patients treated with the drug.
Advertisement
The newly approved drug eteplirsen can be used by only about 13 percent of the patients.
“Every drug for cancer and other serious life-threatening illnesses that the Abigail Alliance pushed for much earlier approval of in our 15- year history is now approved by the FDA”, said Burroughs. You may recall that Sarepta shares also skyrocketed in June following related FDA news, but it may not be the trails the drug maker is blazing with its drug.
The FDA granted Exondys 51 fast track designation, which is a designation to facilitate the development and expedite the review of drugs that are meant to treat serious conditions and that demonstrate the potential to address an unmet medical need.
“We had the feeling we were screaming for the life of these boys and no one could hear us”, said Debra Miller, founder of CureDuchenne of Newport Beach, Calif., one of three patient groups that helped fund Sarepta’s clinical trial.
The FDA, in its approval, also granted Sarepta a Pediatric Review Voucher to speed drug approval, as the company is developing follow-on drugs to Exondys 51.
The Food and Drug Administration approved a controversial muscular dystrophy drug Monday, ignoring the advice of its advisers and delighting families and advocates who had campaigned hard for its approval. The 52 week high Sarepta Therapeutics’s shares have peaked at is 56.18 whilst the 52 week low for the company’s shares is 8.
Advertisement
It’s the first FDA approval for the degenerative condition. The rare genetic disorder, which is the most common for of muscular dystrophy, is known for its progressive muscle deterioration and weakness. For example, Muscular Dystrophy UK requested in April that it accelerate plans to proceed with a marketing application in the EU. The label makes clear that clinical benefit was not established during trials and indicates, as is customary with accelerated approvals, that continued approval in the indication may be contingent upon verification of clinical benefit in confirmatory trials. And not just because they hope to keep taking the drug. The drug’s boosters cited a huge unmet need for such a treatment and enough evidence of its effectiveness to justify approving the drug for patients with no options. “Ultimately, it appears to us that the FDA bowed to external pressure from patient advocates and others who demanded that a safe and potentially efficacious drug be made available”. “A company’s business and financial matters are external to the FDA and do not play a role in the decision to approve or to deny approval of a drug product”, Walsh said in a statement. The FDA encourages companies to develop drugs for orphan diseases by giving them special tax credits and extending the amount of time that companies are able to sell them exclusively, without generic competition.
