Avidity Biosciences' exon skipping drug for an uncommon form of Duchenne muscular dystrophy improved or stabilized functional outcomes at one year. The California biotech company previously announced that it was filing for accelerated approval with the FDA by the end of the year, and it hopes the new data can bolster its case. Avidity’s del-zota is an exon 44 skipping treatment, which could be used for around 6% of people with Duchenne muscular dystrophy. In a Phase 1/2 trial and subsequent extension study, Avidity followed 17 participants for one year. Some initially received a 10 mg/kg regimen while others received a 5 mg/kg regimen, which is the dose that Avidity
advanced. In an open-label extension study, all patients were given the 5 mg/kg dose, which was administered every six weeks, even if they received the higher dose in the Phase 1/2 trial. |