FDA accepts Intellia's lonvo-z application with priority review status

Intellia Therapeutics (Nasdaq: NTLA) announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lonvo-z, a CRISPR-based therapy for hereditary angioedema (HAE), and granted it Priority Review status.

The FDA set a Prescription Drug User Fee Act (PDUFA) target action date of March 10, 2027. The agency also advised Intellia that it does not currently plan to hold an advisory committee meeting to discuss the application.

If approved, Intellia claims lonvo-z would be the first in vivo CRISPR-based therapy and the only one-time treatment for HAE.

The BLA is supported by data from Intellia's Phase 3 HAELO clinical trial, which enrolled 80 patients in nine months. The trial evaluated a one-time 50 milligram dose of lonvo-z in adults and adolescents aged 16 and older with Type 1 or Type 2 HAE.

The trial met its primary and all key secondary endpoints, showing an 87% reduction in mean monthly attacks compared with placebo during the efficacy evaluation period from weeks 5 to 28. Additionally, 62% of patients in the lonvo-z group were attack free and HAE therapy free during the six-month evaluation period, compared with 11% in the placebo group.

The most common adverse events reported in the lonvo-z group included infusion-related reactions, headache, fatigue, back pain, and upper respiratory tract infection. All treatment-emergent adverse events were described as mild or moderate, with no serious adverse events reported in the lonvo-z arm as of the February 10, 2026 data cutoff.

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