BLA accepted with Priority Review for Intellia's lonvo-z in hereditary angioedema
- Sponsor
- Intellia Therapeutics $NTLA
- Drug
- lonvoguran ziclumeran
- Indication
- hereditary angioedema (HAE)
FDA accepted Intellia Therapeutics' BLA for lonvoguran ziclumeran (lonvo-z), an in vivo CRISPR one-time treatment for hereditary angioedema, with Priority Review; PDUFA March 10, 2027.
What this means
Hereditary angioedema (HAE) is a rare genetic disease that causes sudden, severe swelling attacks in the skin, airway, and gut. Current treatments either stop attacks as they happen or must be taken regularly to prevent them. Lonvoguran ziclumeran (lonvo-z) from Intellia Therapeutics is designed to be a single, one-time infusion that uses CRISPR gene editing to permanently switch off a gene in the liver that drives these attacks. The FDA accepted the application for review on Sept 8, granted it Priority Review, and set a decision date of March 10, 2027. FDA also said it is not currently planning to convene an advisory committee to discuss the application. Pivotal support is the global Phase 3 HAELO trial, which read out positive topline data in April 2026. If approved, lonvo-z would be the first in vivo CRISPR-based therapy and the only one-time treatment for HAE.