FDA gives Denali accelerated approval for rare disease drug

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The FDA granted accelerated approval to Denali Therapeutics’ rare disease drug Avlayah, bucking a recent trend of rejections in the space that has put the agency under political pressure.

Avlayah, whose generic name is tividenofusp alfa, will be able to treat patients with Hunter syndrome, a rare genetic disorder that manifests in childhood, who weigh at least 5 kg “prior to advanced neurologic impairment,” according to a Denali press release. It comes with a boxed warning for hypersensitivity (including anaphylaxis) that’s consistent with Avlayah’s drug class, Denali said. The company also received a rare pediatric disease priority review voucher.