Shots:
- FDA has granted accelerated approval to Avlayah, an enzyme replacement therapy (ERT), for the treatment of presymptomatic or symptomatic MPS II pediatric pts (≥5kg), along with a RPD PRV to Denali; US availability is expected shortly
- Approval was based on the P-I/II trial of Avlayah (QW) in 47 pts, incl. ERT-naïve (n=15) & previously treated (n=32), with CSF HS reduction as a surrogate endpoint, showing a 91% decrease & 93% pts reaching normal levels at Wk. 24; data were published in The NEJM
- Avlayah is being evaluated in the ongoing global P-II/III (COMPASS) trial in pts, incl. young adults living with MPS II, to generate confirmatory evidence & support global regulatory filings
Ref: Denali Therapeutics | Image: Denali Therapeutics |Press Release
Related News: Denali Therapeutics Announces Publishing of P-I/II Study of Tividenofusp Alfa (DNL310) in NEJM for Hunter Syndrome (MPS II)
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