Intellia's one-shot HAE gene editor gets priority FDA review, decision due March 2027

Intellia Therapeutics could have the first approved in vivo CRISPR treatment for hereditary angioedema (HAE) by March 2027. The US Food and Drug Administration has accepted its biologics licence application for lonvoguran ziclumeran, also known as lonvo-z and previously NTLA-2002, and granted priority review. The agency's target action date under the Prescription Drug User Fee Act is 10 March 2027.

The company disclosed the timetable in materials prepared for an investor event. According to those materials, the FDA does not at present intend to convene an advisory committee on the application. That stance is not fixed and could change as the review proceeds.

Lonvo-z is designed as a single treatment that edits genes inside the body. The investor materials also cover positive results from the Phase 3 HAELO trial. The material reviewed gave no detailed efficacy or safety figures, so CompePharma is not reporting any. Intellia also described commercial preparations under way ahead of a possible approval, but no information on price, launch timing or patient access was available.

By way of context, HAE treatment is currently built largely around therapies taken on an ongoing basis. A one-time option would therefore enter the market on a very different footing. How it would be priced and positioned with payers has not been set out.