Posted by Michael Wonder on 22 Sep 2016
FDA commissioner calls for Sarepta clinical trial to be retracted
22 September 2016 - In an unusual development, the US FDA Commissioner Dr. Robert Califf has indicated that a clinical trial about a newly approved medicine for patients with Duchenne muscular dystrophy was “misleading” and should be retracted.
The medicine, which was developed by Sarepta Therapeutics, is the first to be approved for the rare genetic disease, which causes steady muscle deterioration, eventually confining boys to wheelchairs and condemning them to an early death.
The medicine, eteplirsen (Exondys 51) was approved despite a rancorous, behind-the-scenes dispute among top FDA officials over the extent to which the medicine can actually benefit patients.
