Thursday, November 14, 2013
FDA Says NDA Filing for Eteplirsen 'Premature'
The U.S. Food and Drug Administration (FDA) has called Sarepta Therapeutics’ application for a new Duchenne muscular dystrophy (DMD) drug “premature.” Citing “considerable doubt” raised by the data, the FDA has asked for additional research on eteplirsen, with perhaps different clinical endpoints, combined endpoints, and different DMD subpopulations for a confirmatory clinical study.
The FDA also questioned the use of dystrophin as a valid biomarker, and the supportive clinical efficacy assessed on the 6-minute walk test (6MWT) in the Phase IIb clinical study of eteplirsen. They emphasized the need for a placebo-controlled study. Sarepta said in a public conference call, that these requests will delay the initiation of dosing in the eteplirsen confirmatory study until at least the second quarter of 2014. They have scheduled a follow-up meeting this month with the FDA to discuss the confirmatory study design.
The FDA told Neurology Today it was unable to comment on investigational new drug applications.
“We are very disappointed with the FDA's decision to reconsider their openness to a potential NDA filing based on our current data and the resultant impact this change may have on our efforts to achieve an earlier approval of eteplirsen," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We strongly believe in the potential of eteplirsen to address a serious unmet medical need in DMD and we are committed to its development."
See our previous coverage of eteplirsen: http://bit.ly/NJfgvv. Stay tuned for a full story in the upcoming issue of Neurology Today.