Denali Therapeutics Announces Publishing of P-I/II Study of Tividenofusp Alfa (DNL310) in NEJM for Hunter Syndrome (MPS II)

Shots: The NEJM published results from the P-I/II study of Denali Therapeutics’ tividenofusp alfa (DNL310) for the treatment of Hunter syndrome (MPS II) The study evaluated treatment in 47 ERT-naïve (n=15) and previously treated (n=32) study participants (aged 0.3–13 [median, 5] years) demonstrating reduced levels of mean CSF levels of HS, mean urine HS levels,…

ByDipanshu DixitJanuary 2, 2026

Denali Therapeutics Reports the US FDA’s BLA Acceptance and Priority Review of Tividenofusp Alfa for Hunter Syndrome

Shots: The US FDA has accepted BLA & granted priority review to tividenofusp alfa for the treatment of Hunter syndrome (MPS II), with PDUFA target action date of Jan 5, 2026 BLA was supported by a P-I/II trial of tividenofusp alfa in 47 MPS II pts, as Denali prepares for its US commercial launch. It…

ByRidhi RastogiJuly 8, 2025

REGENXBIO Reports the US FDA’s BLA Acceptance and Priority Review of RGX-121 for Treating Mucopolysaccharidosis II

Shots: The US FDA has accepted BLA & granted priority review to RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II or Hunter syndrome), with PDUFA action date of Nov 09, 2025 As per Jan 2025 partnership, NS Pharma (Nippon Shinyaku’s subsidiary) will commercialize RGX-121 in the US post approval while REGENXBIO will handle…

ByRidhi RastogiMay 14, 2025

Denali Therapeutics Announces Publishing of P-I/II Study of Tividenofusp Alfa (DNL310) in NEJM for Hunter Syndrome (MPS II)

Denali Therapeutics Reports the US FDA’s BLA Acceptance and Priority Review of Tividenofusp Alfa for Hunter Syndrome

REGENXBIO Reports the US FDA’s BLA Acceptance and Priority Review of RGX-121 for Treating Mucopolysaccharidosis II

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