Announcement
Intellia Therapeutics (NASDAQ: NTLA) reported that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for lonvoguran ziclumeran (lonvo‑z) for the treatment of hereditary angioedema (HAE) and granted Priority Review.
Regulatory Timeline
The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of 10 March 2027 and indicated that it does not intend to convene an advisory committee to discuss the application.
Clinical Data
The BLA is supported by the Phase 3 HAELO trial, which enrolled 80 patients over nine months and evaluated a single 50 mg dose of lonvo‑z in adults and adolescents aged 16 years and older with Type 1 or Type 2 HAE. The trial met its primary endpoint and all key secondary endpoints, showing an 87 % reduction in mean monthly attacks for lonvo‑z versus placebo during weeks 5‑28. Additionally, 62 % of patients receiving lonvo‑z were attack‑free and free from HAE therapy for the six‑month efficacy evaluation period, compared with 11 % in the placebo arm. As of the data cutoff on 10 February 2026, all patients who received lonvo‑z at baseline or in crossover after week 28 remained free from long‑term prophylaxis therapy.
Safety Profile
The most frequent treatment‑emergent adverse events occurring more often in the lonvo‑z group than in placebo were infusion‑related reactions, headache, fatigue, back pain, and upper respiratory tract infection. All reported events were classified as mild or moderate, and no serious adverse events were observed in the lonvo‑z arm.
Market Reaction
Following the announcement, Intellia’s shares increased approximately 8 % on Tuesday, 8 September 2026.