The FDA caused major controversy by approving the drug Exondys 51 from Sarepta Therapeutics for treating Duchenne muscular dystrophy over the recommendations of a scientific panel it convened that had voted 7-3 against approval.
Is this contentious Sarepta approval serve as a sign of how the FDA will deal with stem cells moving forward?
Will the FDA approval, which was based on very limited, unclear data and in large part due to pressure from some patient advocates, serve as a warning to those of us hoping the agency will not yield to political or other pressure in weakening oversight of investigational stem cell therapies?
Newly released internal FDA emails show that Sarepta approval was enormously controversial within the agency itself. A top FDA scientist challenged the agency’s decision in strong terms, noting that “Sarepta was unable to reproduce its own findings and there was “no way to reach a rational conclusion” a “reasonably likely” clinical benefit could be predicted.”
I wonder if similar debates within the FDA could be going on right now related to its four draft guidances on stem cells and regenerative medicine. Ultimately could the agency leadership make judgment calls without data in effect green lighting stem cell clinics to continue offering unproven stem cell therapies and putting patients at grave risk?
If I had to guess I would say optimistically that this is unlikely to happen, particularly given the disastrous case studies the FDA heard at the meetings it called on stem cells. For instance, we heard more about the patient Jim Gass developing a spinal tumor and the breaking story of at least three women reportedly being blinded by a stem cell clinic in Florida. A man in the audience reportedly wore a sign around his neck saying he was blinded by a stem cell clinic. I have heard through the grapevine that more bad news is coming from Florida on the stem cell clinic front.
One way or another the FDA should say something about its draft guidances on stem cells by early 2017. The number of stem cell clinics appears to be continuing to shoot up. The FDA can be more efficient in reviewing stem cell products in development, but it all has to remain data-centric.
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