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Drug reviews are a frustratingly opaque process, so absent subpoena power, one cannot fully discount the possibility that Regenxbio’s Hunter syndrome gene therapy data were weak, totally screwed up, or some combination of both. If that’s what happened, the Food and Drug Administration’s decision to reject the therapy was justifiable.
But it’s hard to ignore a broader and more troubling trend: The Regenxbio treatment, called RGX-121, joins a growing list of cell and gene therapies for rare diseases that have been rejected or delayed since the appointment of Vinay Prasad as the head of the FDA center that regulates them.
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