SILVER SPRING
Avlayah granted accelerated approval for Hunter syndrome
- Sponsor
- Denali Therapeutics Inc. $DNLI
- Drug
- Avlayah · tividenofusp alfa-eknm
- Indication
- Hunter syndrome (MPS II)
FDA granted accelerated approval to Denali Therapeutics' Avlayah (tividenofusp alfa-eknm) for the treatment of Hunter syndrome (MPS II).
What this means
Avlayah is an enzyme replacement therapy that helps break down harmful sugar molecules that build up in the body of patients with Hunter syndrome, a rare genetic disorder. Today FDA granted accelerated approval to Avlayah based on early evidence suggesting it may benefit patients. This decision provides a new treatment option while Denali continues studies to confirm the drug's long-term effectiveness and safety.