FDA accepts BLA for del-zota in DMD44 with priority review

Novartis/Del-zota (delpacibart zotadirsen)

FDA accepted BLA for del-zota in DMD with exon-44-amenable mutations with priority review. Phase 3 SAFARI44 confirmatory trial underway.

• BLA includes Phase 1/2 EXPLORE44 trial data
• Priority review and accelerated approval pathway
• First-in-class antibody-oligonucleotide conjugate (AOC)
• Phase 3 SAFARI44 trial for full approval support
• Targets exon-44-skipping for ~10% of DMD patients