Avidity Biosciences granted Breakthrough Therapy Designation for exon-44 skipping therapeutic, del-zota

CureDuchenne was an early investor in Avidity Biosciences in 2018, and congratulates the company for receiving Breakthrough Therapy Designation from the US FDA for delpacibart zotadirsen (abbreviated as del-zota), their exon 44-skipping agent for the treatment of Duchenne. 

Why is this important: 

The FDA grants Breakthrough Therapy Designations to speed up the development and review of drugs for serious conditions when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over an available therapy on clinically significant endpoints. This designation offers several benefits to Avidity, including enhanced interactions with key senior-level staff the FDA, as well as Rolling and Priority Review eligibility, which will can significantly reduce the time the FDA uses for making an approval decision.

What’s next:

Avidity is planning to submit materials to the FDA for accelerated approval of del-zota by the end of 2025. 

Link to press release: https://investors.aviditybiosciences.com/2025-07-23-Avidity-Biosciences-Receives-FDA-Breakthrough-Therapy-Designation-for-Delpacibart-Zotadirsen-del-zota-for-the-Treatment-of-DMD-in-People-with-Mutations-Amenable-to-Exon-44-Skipping