FDA accepts BLA for Lonvo-z with Priority Review for HAE

Intellia Therapeutics announced that the U.S. Food and Drug Administration has accepted its Biologics License Application for lonvoguran ziclumeran, referred to as Lonvo-z. The FDA also granted Priority Review for the application, setting a Prescription Drug User Fee Act target action date of March 10, 2027.

The company noted that the FDA currently does not plan to convene an advisory committee to discuss the application. Lonvo-z is being developed as a treatment for hereditary angioedema (HAE). If approved, Intellia states it would be the first in vivo CRISPR-based therapy globally and the only one-time treatment for HAE.

The BLA submission is supported by data from the global Phase 3 HAELO clinical trial. This trial assessed a single 50 milligram dose of Lonvo-z in 80 adult and adolescent patients aged 16 and older with Type 1 or Type 2 HAE. The study met its primary and all key secondary endpoints, showing an 87% reduction in mean monthly attacks compared to placebo during the efficacy evaluation period from week 5 to week 28. Additionally, 62% of patients receiving Lonvo-z were attack-free and HAE therapy-free for this six-month period, compared to 11% in the placebo group.

Safety data from the trial indicated favorable tolerability for Lonvo-z. The most common treatment-emergent adverse events in the Lonvo-z group, occurring more frequently than in the placebo group during the primary observation period (infusion through week 28), included infusion-related reactions, headache, fatigue, back pain, and upper respiratory tract infection. All reported adverse events were mild or moderate, with no serious adverse events noted in the Lonvo-z arm.