Del-zota Priority Review granted for Duchenne muscular dystrophy amenable to exon 44 skipping
- Sponsor
- Novartis AG $NVS
- Drug
- del-zota · delpacibart zotadirsen
- Indication
- Duchenne muscular dystrophy amenable to exon 44 skipping
FDA accepted Novartis's BLA for del-zota (delpacibart zotadirsen) under Priority Review, targeting the Accelerated Approval pathway for people with Duchenne muscular dystrophy amenable to exon 44 skipping. Filing rests on Phase 1/2 EXPLORE44 and EXPLORE44-OLE data.
What this means
Duchenne muscular dystrophy is a fatal genetic muscle-wasting disease with only a handful of approved therapies, and none for the roughly 8 percent of patients whose mutation would benefit from skipping exon 44 in the dystrophin gene. Del-zota is a targeted therapy that uses an antibody to carry a small nucleic acid into muscle cells, where it tells the cell to skip that broken piece and produce a shorter but functional dystrophin protein. FDA has accepted Novartis's application and granted Priority Review under the Accelerated Approval pathway, based on Phase 1/2 data from the EXPLORE44 studies. Del-zota came into Novartis through the Avidity Biosciences acquisition.