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biotech/news//STAT
The FDA approved a new medicine from Denali Therapeutics for a condition called Hunter syndrome.
The FDA approved Denali Therapeutics' Avlayah for Hunter syndrome despite recent tough standards.
KEY POINTS
Denali's Avlayah approval contrasts with Regenxbio's gene therapy rejection for the same condition.
The FDA cited insufficient clinical data when rejecting Regenxbio's Hunter syndrome gene therapy.
Advocates feared Avlayah would be rejected due to a trend of increased FDA scrutiny.
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.
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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.