FDA tentatively approves first drug for muscular dystrophy

Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.

But most recently, the indications leaned toward approval when a prominent critic within the FDA left the agency for another job, which seemed to improve chances of approval. Sarepta is still required to conduct a clinical trail to confirm the drug’s benefits, and the FDA could withdraw its approval of the drug if the trial fails to show clinical benefits. Those scant data compared patients on eteplirsen to a “historical control”-previous data on patients deemed to have similar characteristics to those who took the drug-not to patients receiving a placebo in the study, which the FDA prefers”. Sarepta shares soared then, as Farkas’ departure was seen by investors to pave the way for approval. Some staffers said there was little evidence the drug worked. It also touched off a barbed debate between those who applauded the move as giving hope to desperate patients – and those who warned it would backfire since there is no clear evidence the drug works. It’s “an X-linked recessive inheritance pattern and is passed on by the mother, who is referred to as a carrier of the gene”, according to the Muscular Dystrophy Association.

The disease is so rare that the FDA considers it an “orphan disease, ” or one that isnt common enough to attract many drug developers.

In 2012 Congress gave the FDA extra tools and a mandate to get new drugs for deadly diseases to patients more quickly.

At the conclusion of the epic meeting, which included 52 presenters – mostly patients, family members and clinicians – during a gut-wrenching three-hour open public hearing, panelists concluded that Sarepta failed to prove its case statistically. And Sarepta and Duchenne patients wouldn’t have to wait years for results from a larger trial.

The drug treats a subset of patients with Duchenne muscular dystrophy, a rare, progressive genetic disorder that hampers muscle movement, eventually killing most sufferers by age 30.

Accelerated approval was granted to the new drug based on clinical studies that found an increase in the dystrophin protein among some people treated with Exondys 51.

At a meeting in April, the agency walked a hard high-wire act, visibly struggling to strike the balance between respecting the views of parents and boys who attributed their health to the drugs while also pointing out that the data showed scant evidence of the positive effects reported.

Some health advocates criticized the FDAs decision. But regulators faced an enormous outcry from patient groups and physicians seeking access to the drug.

“It sets a risky precedent if the FDA is going to start approving drugs that arent compared to anything, ” Zuckerman said.

SRPT surged 76.6% Monday on the FDA’s approval. The drug could be available to patients before the end of the year.

“Im really overwhelmed, ” McLinn said.

“Today has been a long time in the making”, said MDA President and CEO Steven M. Derks. “In her capacity as acting chief scientist, Dr. Borio also conveyed her own views on the dispute, stating among other things that she does not believe the available data and information support accelerated approval of eteplirsen”.

Eteplirsen was initially set to come before the FDA’s Peripheral and Central Nervous System Drugs advisory committee (adcom) in January, where it faced a hostile reception from FDA reviewers. BioMarin was forced to announced this spring that it would end pursuit of approval of its drug, Kyndrisa (drisapersen).