The dominos are starting to fall: Avidity/Novartis has submitted their DMD drug to the FDA
Hi friends! While this is DMD news, I actually think it’s a huge milestone for those of us following Del-brax.
Avidity/Novartis officially submitted the BLA for their DMD drug, Del-zota, meaning it’s now officially in the FDA’s hands for review (the FDA has ~ 60 days to accept, and then the six-month Priority Review timeline starts).
Here’s why this matters for us: by the time Del-brax is submitted, it won’t be the FDA’s first AOC rodeo.
Del-zota and Del-brax are built on the same AOC platform, and a BLA is about a lot more than just whether a drug works or not. The FDA also reviews the manufacturing process, quality controls, preclinical package, and all of the platform-level science behind the therapy. (It’s like a massive 1000+ page submission package.)
So while Del-zota and Del-brax are different drugs, many of those pieces are shared across the AOC platform.
Now back to FSHD specifically. We just got high-level positive Phase 2b biomarker data, and before the acquisition Avidity announced the accelerated approval pathway was open for Del-brax. Novartis has since reiterated that pathway remains on the table while they evaluate the full dataset. 🤞
If those data support a filing, Del-brax could realistically be the next AOC BLA, potentially later this year (wishful thinking but you never know) or early next!
That’s what makes this news so exciting. By the time Del-brax lands on the FDA’s desk, it won’t be the FDA’s first time seeing this novel drug platform. They’ll already have spent months reviewing the platform through Del-zota, and Avidity/Novartis will have gone through the whole BLA process once already and can fold those learnings into the Del-brax application.
There’s still a lot we don’t know. But it really feels like the dominoes are starting to fall. If everything keeps moving in the right direction, it’s kind of wild to think we could have an IV in our arms sometime in mid-late 2027!