Lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002) - (HAELO)

September 8, 2026: Intellia Therapeutics (NASDAQ: NTLA) said the FDA accepted the BLA for lonvo-z (lonvoguran ziclumeran; formerly NTLA-2002) for hereditary angioedema (HAE) and granted Priority Review, with a PDUFA target action date of March 10, 2027. FDA also said it is not currently planning an advisory committee.

The filing is supported by the global Phase 3 HAELO trial in 80 adults and adolescents age 16+ with Type 1 or Type 2 HAE, evaluating a one-time 50 mg dose. HAELO met the primary and all key secondary endpoints, showing an 87% reduction in mean monthly attacks versus placebo during weeks 5-28 (p<0.0001). Also, 62% of lonvo-z patients were attack-free and HAE-therapy-free during the 6-month efficacy period versus 11% on placebo (p<0.0001). As of the February 10, 2026 cutoff, all patients dosed at baseline or after crossover remained free from long-term prophylaxis therapy.

Safety was described as favorable: the most common adverse events occurring more often than placebo were infusion-related reactions, headache, fatigue, back pain, and upper respiratory tract infection; all were mild or moderate, with no serious adverse events in the lonvo-z arm.