Zeleciment rostudirsen

Duchenne muscular dystrophy is an X-linked dystrophinopathy associated with progressive weakness and respiratory, cardiac, and skeletal complications. A systematic review estimated birth prevalence at 19.8 per 100,000 live male births. A survival meta-analysis estimated median life expectancy at 28.1 years among those born after 1990. In a U.S. analysis with 782 matched pairs, mean annual healthcare costs were US$103,826.05 for the DMD cohort and US$3,225.30 for the non-muscular-dystrophy cohort. These estimates describe different populations and should not be combined into a product-specific economic estimate.

The DELIVER registrational expansion cohort included 32 participants, with 24 allocated to z-rostudirsen and 8 to placebo. At six months, mean absolute dystrophin was 5.46% of normal after muscle-content adjustment and 2.87% without adjustment. A post-hoc functional comparison used 21 treated participants and 18 pooled placebo participants. Reported differences were 0.04 rise/sec for TTR velocity and 0.2 m/sec for 10MWR velocity, both with nominal p<0.05; the NSAA difference was 2.1 points with nominal p=0.0894. No related serious TEAEs were reported in the registrational cohort. Related serious events occurred in extension participants, and higher-dose events included acute kidney injury, thrombocytopenia, and pancytopenia.

FDA acceptance of the BLA and Priority Review do not establish approval. The reported target action date is January 21, 2027. The application uses dystrophin as a surrogate endpoint; the available functional comparisons remain exploratory. FORZETTO is the confirmatory Phase 3 trial. No product-specific budget impact model or acquisition price is available in the evidence reproduced here.