FDA Grants Priority Review for Intellia Therapeutics' CRISPR-Based Therapy for Hereditary Angioedema

The U.S. Food and Drug Administration (FDA) has accepted Intellia Therapeutics' Biologics License Application (BLA) for lonvoguran ziclumeran (lonvo-z) and granted it Priority Review. If approved, lonvo-z would be the world's first in vivo CRISPR-based therapy and the only one-time treatment for hereditary angioedema (HAE), a rare and unpredictable disease. The BLA is supported by positive data from the global Phase 3 HAELO clinical trial. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of March 10, 2027, and is not currently planning an advisory committee meeting for the application.