- Parent organizers of the grass roots campaign to get the regulator to approve the drug for children afflicted with Duchenne muscular dystrophy sent a letter to FDA Commissioner Margaret Hamburg today urging her to direct the agency to expedite its guidance to Sarepta Therapeutics (SRPT +4%) so it may file its NDA. The company has been waiting for said guidance for 16 months.
- Once submitted, the organizers want an accelerated review of the application. Currently, there are no approved medications for the disease that afflicts 1 in 3,500 boys.
- The campaign, which was a petition to the White House, garnered over 105,000 signatures.