Rare diseases
With two decisions originally scheduled for this week already announced, including BridgeBio’s approval in ATTR-CM, the regulator has just one PDUFA on its plate this holiday week.
Despite the death, the FDA has allowed Neurogene to forge ahead with the Phase I/II Rett syndrome trial, but using only the lower 1E15 vg dose of its investigational gene therapy NGN-401.
A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications.
After failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells investors that it’s evaluating options for the program.
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.
Analysts appear optimistic for Intellia’s gene editor nex-z, which showed a greater serum TTR reduction than Alnylam’s Amvuttra.
Bluebird has just two quarters until it’s out of cash. Executives are looking for financing to extend that runway to a projected breakeven point before the end of 2025, with analysts worried they won’t make it.
Following strong treatment response data for Adaptimmune’s lete-cel, the biotech is planning to initiate a rolling BLA submission to the FDA, set to start by the end of 2025.
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene therapy to be delivered directly to the brain.
Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated at a lower dose and showed promising efficacy outcomes.
PRESS RELEASES