The Food and Drug Administration on Wednesday approved a new medicine from Denali Therapeutics for a condition called Hunter syndrome, a notable decision by the agency as it has recently taken tougher stances on rare disease drugs.
Last month, the FDA rejected a Hunter syndrome gene therapy from Regenxbio, saying that the company needed to produce more clinical data, a higher standard of evidence that could take the company years to meet.
The gene therapy’s denial — on top of other FDA rejections of rare disease therapies and signals that officials were scrutinizing the drugs more closely — left advocates worried about the fate of Denali’s drug, called Avlayah.
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