FDA accepts Intellia's BLA for lonvo-z in hereditary angioedema, grants priority review

Intellia Therapeutics said on September 8, 2026 that the FDA has accepted the Biologics License Application for lonvoguran ziclumeran (lonvo-z) and granted Priority Review, with a PDUFA target action date of March 10, 2027¹.

The company noted that the FDA has advised it is not currently planning to hold an advisory committee to discuss the application¹. Intellia said that, pending approval, lonvo-z would be the world's first in vivo CRISPR-based therapy and the only one-time treatment for HAE¹.

The filing is based on results from the Phase 3 HAELO trial. The study enrolled 80 patients in nine months and tested a one-time 50 milligram dose of lonvo-z in adults and adolescents 16 and older with Type 1 or Type 2 HAE.¹ The trial reached its primary and all key secondary endpoints, showing an 87% reduction in mean monthly attacks versus placebo during weeks 5 through 28, with a p-value below 0.0001¹. In addition, 62% of patients on lonvo-z were free of attacks and HAE therapy during the six-month evaluation period, compared with 11% on placebo¹. As of a February 10, 2026 cutoff, every patient who received lonvo-z, either at baseline or after crossover following week 28, remained off long-term prophylaxis¹.

On safety, the most common adverse events more frequent in the lonvo-z group through week 28 were infusion reactions, headache, fatigue, back pain, and upper respiratory infection, all mild or moderate, with no serious adverse events in that arm¹.

Lonvo-z, formerly NTLA-2002, already holds Orphan Drug and RMAT designations from the FDA, Innovation Passport from the UK's MHRA, PRIME designation from the European Medicines Agency, and Orphan Drug Designation from the European Commission¹.