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, Serum neurofilament light (NfL) chain levels at wk. 49 and NfL at wk. 153 were reduced by 91%, 88%, 21% and 76% respectively; the study also showed stabilization or improvement in adaptive behavior, cognition and hearing, and normalization of liver volume
- The BLA for tividenofusp alfa is under US FDA Priority Review (PDUFA: Apr’5, 2026). In addition, the therapy has received BTD & RPDD
Ref: Denali Therapeutics | Image: Denali | Press Release
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