← All approvals · Rare & genetic · 2026
Avlayah (tividenofusp alfa-eknm)
The FDA approved Avlayah (tividenofusp alfa-eknm) on March 24, 2026 to treat certain individuals with Hunter syndrome (Mucopolysaccharidosis type II or MPS II).
- Brand name
- Avlayah
- Generic name
- tividenofusp alfa-eknm
- FDA approval date
- March 24, 2026
- Approved use (FDA wording)
- To treat certain individuals with Hunter syndrome (Mucopolysaccharidosis type II or MPS II)
- Company
- Denali Therapeutics Inc..
- Application
- BLA761485
- Dosage form / route
- INJECTION · POWDER,FOR SOLUTION
- Drug class (EPC)
- Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme [EPC]
- Boxed warning on label
- Yes (see the FDA label)
- Category on this site
- Rare & genetic
Indications and usage, from the FDA label
AVLAYAH is indicated for the treatment of neurologic manifestations of Hunter syndrome (Mucopolysaccharidosis type II, MPS II) when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment. This indication is approved under accelerated approval based on the reduction of cerebrospinal fluid heparan sulfate [see Clinical Studies (14) ] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Limitations of Use AVLAYAH is not recommended for use in combination with other enzyme replacement therapies for the treatment of Hunter syndrome. AVLAYAH …