Biopharmaceutical company Avidity Biosciences Inc (Nasdaq:RNA) announced on Wednesday its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the United States.
Under an FDA-authorised treatment protocol, Avidity will provide del-zota to eligible boys and men with DMD44 through participating healthcare providers. Enrolment is anticipated to begin by year end, and participants in EXPLORE44-OLE will have the option to transition to the MAP as they complete 2 years of treatment.
Avidity says that it aligned with the FDA on a path forward for a BLA submission for del-zota following an October 2025 pre-BLA meeting, with the submission planned for 2026 for accelerated approval.
Participants will transition to commercial drug supply upon future potential FDA approval and product availability.
DATROWAY receives US priority review for first-line metastatic triple negative breast cancer
Lupin launches Dasatinib tablets in US market
Natera submits Signatera CDx PMA to FDA for bladder cancer use
Pharming receives FDA complete response letter for paediatric Joenja application
Trace Biosciences' IND application for nerve-specific imaging agent approved by FDA
Frontage expands early phase clinical research capabilities across US and China
MicuRx Pharmaceuticals' IND application for MRX-5 cleared by FDA
FDA approves Tenpoint Therapeutics' YUVEZZI as first dual-agent eye drop for presbyopia
Summit Therapeutics' BLA for ivonescimab in EGFR-mutated NSCLC accepted by FDA
WuXi Biologics collaborates with Sinorda Biomedicine for antibody development
Biogen's litifilimab receives FDA Breakthrough Therapy Designation for CLE
Glaukos receives FDA approval for repeat administration of iDose TR
Guerbet's contrast agent Elucirem approved by European Commission in children from birth
Spine Innovation's LOGIC Titanium Implant System receives US FDA 510(k) market approval