• FDA sets Prescription Drug User Fee Act (PDUFA) date of March 10, 2027
• Positions lonvo-z to be the world’s first in vivo CRISPR-based therapy and the only one-time HAE treatment, if approved

Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, announced the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lonvo-z and granted the BLA Priority Review with a PDUFA target action date of March 10, 2027. Additionally, FDA has advised the company that it is not currently planning to hold an advisory committee to discuss the application. If approved, lonvo-z would be the world’s first in vivo CRISPR-based therapy and the only one-time treatment for HAE.

“Today marks an important milestone for the patients we are committed to serving and for Intellia’s pioneering work in the field of in vivo gene editing,” said John Leonard, M.D., Intellia President and Chief Executive Officer. “Backed by compelling Phase 3 data, we believe lonvo-z could fundamentally change the way HAE is treated and are excited by its potential to become the world's first approved in vivo CRISPR-based therapy. With the FDA’s Priority Review underway, our team is well prepared to deliver this one-time treatment to patients who are waiting for new options.”

Joshua Jacobs, M.D., Medical Director, Allergy and Asthma Clinical Research, Inc., and a HAELO trial investigator, added, “HAE is an unpredictable disease that can be responsible for profound disability and place patients at risk for fatal attacks. Today’s announcement is exciting because it advances us one step closer to potentially having a one-time treatment option available for patients who continue to be burdened by this chronic disease.”

The BLA is supported by positive data from Intellia’s global Phase 3 HAELO clinical trial, which was fully enrolled with 80 patients in just nine months and was designed to evaluate the efficacy and safety of a one-time 50 milligram dose of lonvo-z in adults and adolescents aged 16 years and older with Type 1 or Type 2 HAE. HAELO met its primary and all key secondary endpoints, demonstrating an 87% reduction (p<0.0001) in mean monthly attacks for lonvo-z compared with placebo during the efficacy evaluation period (weeks 5 to 28). In addition, 62% of patients in the lonvo-z arm were entirely attack free and HAE therapy free for the six-month efficacy evaluation period, compared with 11% of patients in the placebo arm (p<0.0001). As of the February 10, 2026 data cutoff, all patients who received lonvo-z at baseline or in crossover after week 28 remained free from long-term prophylaxis therapy.

Favorable safety and tolerability data were observed for lonvo-z as of the data cutoff. The most common treatment emergent adverse events during the primary observation period (infusion through week 28) that were higher in the lonvo-z group compared to placebo were infusion-related reactions, headache, fatigue, back pain, and upper respiratory tract infection. All reported treatment emergent adverse events were mild or moderate and there were no serious adverse events observed in the lonvo-z arm.