USFDASep 8, 2026, 04:15 PM
Intellia Therapeutics: FDA Accepts BLA for Lonvo-z with Priority Review
AI Summary
Intellia Therapeutics announced the FDA has accepted its Biologics License Application (BLA) for lonvoguran ziclumeran (lonvo-z) for the treatment of hereditary angioedema (HAE), granting it Priority Review with a target action date of March 10, 2027. The FDA has indicated it does not plan to hold an advisory committee meeting for this application. If approved, lonvo-z would represent a significant advancement as the world's first in vivo CRISPR-based therapy and a potential one-time treatment for HAE. Phase 3 trial data demonstrated substantial reductions in HAE attacks and a high percentage of patients remaining attack-free.
Key Highlights
- FDA accepted Biologics License Application (BLA) for lonvoguran ziclumeran (lonvo-z).
- BLA granted Priority Review with a PDUFA target action date of March 10, 2027.
- FDA is not currently planning to hold an advisory committee meeting for the application.
- If approved, lonvo-z would be the world's first in vivo CRISPR-based therapy.
- If approved, lonvo-z would be the only one-time treatment for hereditary angioedema (HAE).
- Phase 3 HAELO trial showed an 87% reduction in mean monthly HAE attacks compared to placebo.
- 62% of patients in the lonvo-z arm were attack-free and therapy-free for six months.
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