Sarepta Therapeutics (SRPT)

[Kallie]

On February 24, 2016, 38 Duchenne researchers and clinicians sent a letter to FDA in support of eteplirsen's data. The clinicians who signed that letter have collectively treated 5,000 Duchenne patients over the course of 15 years. Their scientific and medical evaluation of eteplirsen's data led them to conclude that the most scientifically robust and ethical way forward was for the FDA to grant eteplirsen accelerated approval and follow up with confirmatory trials to ensure efficacy.

On April 16, 2016, 24 Members of the United States Senate, including Senators Elizabeth Warren (D-MA) and Edward Markey (D-MA), sent a letter to FDA urging the agency to use accelerated approval and to take the patient perspective into consideration when reviewing drugs to treat Duchenne. On February 18, 2016, 109 Members of of the United States House of Representatives, including Representatives Keating, Lynch, Capuano, Tsongas, and McGovern sent a letter to FDA expressing similar sentiments.

"I look forward to joining researchers, scientists and Duchenne families in Maryland this week," said Basile. "I share their hope that the FDA will act quickly to approve a safe and effective treatment for Duchenne, and I am proud and grateful to be representing Governor Baker and all Massachusetts Duchenne families at this important meeting."