25.03.16. 

An advisory panel to the Food and Drug Administration voted not to recommend approval of Eteplirsen from Sarepta Therapeutics. Eteplirsen is an exon skipping drug, and is suitable for patients with a mutation amenable to exon 51 skip, which is approximately 13% of patients. 

The panel were deciding whether to approve the drug based on a single small study of 12 patients. 

More than 900 patients, families and caregivers including 42 representatives from the UK, attended the FDA's Adcomm on Monday 25th April to consider granting Accelerated Approval to Sarepta's exon skipping drug, Eteplirsen.

The advisory committee voted 7-3, against granting approval based on evidence of effectiveness for Eteplirsen. Three panel members abstained. The panel chairman, G. Caleb Alexander an associate epidemiology professor at the Johns Hopkins School of Public Health, said: “I felt this wasn’t a well-controlled study,”  

The three members of the panel who abstained on on the final vote, said they were undecided because they were moved by public testimony from parents who believed their children had been helped by the drug.

After the meeting Emily and Alex had the opportunity to speak to Janet Woodcock (pictured), who has the ultimate decision on whether or not to grant some kind of conditional or accellerated approval to Sarepta. 

Read this opinion piece in the Wall Street Journal

Read Alex's blog here