FDA requirements presented to Sarepta Therapeutics for eteplirsen application for Duchenne Muscular Dystrophy
In meeting minutes received last week from a Type B Pre-NDA meeting that took place in September 2014, the FDA provided updated guidance regarding the specific data to be included as part of, or at the time of, Sarepta's NDA submission for eteplirsen for treatament of Duchenne Muscular Dystrophy.
The guidance states that additional data are now required as part of the NDA submission, including the results from an independent assessment of dystrophin images and the 168-week clinical data from study 202. Additionally, the guidance requests more specific data including a minimum duration of safety in new patients exposed to eteplirsen, patient-level natural history data to be obtained by Sarepta from independent academic institutions, and MRI data from a recent study conducted by an independent academic group. The FDA indicated that further discussion with Sarepta "will be necessary to determine what would constitute a complete NDA." Based on these requests, Sarepta plans to submit an NDA by mid-year 2015, pending any additional requests from further discussions with the FDA.
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