Intellia's lonvo-z BLA accepted by FDA with Priority Review for HAE; PDUFA March 10, 2027

Intellia Therapeutics, Inc.

• FDA accepted BLA for lonvo-z (formerly NTLA-2002) for HAE with Priority Review; PDUFA target date March 10, 2027.
• No advisory committee currently planned; if approved, lonvo-z would be first in vivo CRISPR therapy and only one-time HAE treatment.
• Phase 3 HAELO (80 patients): 87% reduction in mean monthly attacks vs placebo (p<0.0001) during weeks 5-28.
• 62% of lonvo-z patients attack-free and therapy-free for 6 months vs 11% placebo (p<0.0001).
• No serious adverse events in lonvo-z arm; most common TEAEs were infusion-related reactions, headache, fatigue, back pain.