Lonvo-z (lonvoguran ziclumeran) for Hereditary angioedema
The FDA is expected to decide on Lonvo-z for Hereditary angioedema by March 10, 2027, under a BLA from Intellia Therapeutics.
What is being decided
Intellia Therapeutics has a BLA under FDA review for Lonvo-z (lonvoguran ziclumeran) in Hereditary angioedema. Would be the first in vivo CRISPR gene editing therapy approved anywhere, given as a single one-time infusion.
Who this decision matters to
Hereditary Angioedema is a rare genetic disorder characterized by sudden, severe swelling (angioedema) of skin and mucous membranes affecting the face, hands, feet, gastrointestinal tract, and potentially the airway. Most cases result from deficiency of C1-esterase inhibitor, a protein that regulates inflammatory pathways. Attacks are unpredictable and can be life-threatening if the airway is affected.
Prevalence: Approximately 1 in 10,000 to 50,000 people; estimated 5,000-10,000 people in the United States. See our full Hereditary Angioedema page for current treatments, recruiting trials, and community resources.
What each outcome would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. CRLs are often about manufacturing or data presentation rather than efficacy, and resubmission is common. Either way, this page updates with the outcome and what it means.
Where Hereditary Angioedema treatment stands today
Research in Hereditary Angioedema is transforming treatment with a diverse array of new approaches targeting different points in the disease pathway. Kallikrein inhibitors, Factor XII inhibitors, and bradykinin receptor antagonists represent multiple strategic approaches to preventing attacks and reducing their frequency and severity. Three newer medicines were FDA approved in 2025, each for people 12 and older. Garadacimab (Andembry), a once-monthly injection under the skin that blocks activated factor XII, was approved in June 2025 to prevent attacks. Sebetralstat (Ekterly), a plasma kallikrein inhibitor taken as tablets by mouth at the first sign of an attack, was approved in July 2025 to treat attacks. Donidalorsen (Dawnzera), an antisense medicine (a drug that lowers the body's production of the protein prekallikrein) injected under the skin every 4 or 8 weeks, was approved in August 2025 to prevent attacks. Long-acting prophylactic therapies offer the promise of sustained protection, while on-demand treatments for acute attacks continue being refined for faster symptom relief. Trials are investigating how to best use these newer options as first-line prophylaxis and how to optimize long-term prophylaxis strategies that maximize quality of life while minimizing medication burden. Specialty centers focused on immunology and rare diseases often have direct access to trial information and can provide comprehensive care coordination tailored to your specific needs.
Meanwhile, 23 Hereditary Angioedema trials are recruiting
Whatever the FDA decides here, research on Hereditary Angioedema does not stop. A few currently enrolling studies, US sites first:
Other drugs Trial Friend tracks for Hereditary Angioedema
Get notified when new hereditary-angioedema trials open or existing trials change status, add sites, or update eligibility.
Our coverage
Frequently asked questions
When will the FDA decide on Lonvo-z?
The FDA's target decision date (PDUFA date) for Lonvo-z in Hereditary angioedema is March 10, 2027, disclosed by Intellia Therapeutics. The agency can act before this date and occasionally runs past it.
What is Lonvo-z being reviewed for?
Intellia Therapeutics submitted a BLA for Lonvo-z in Hereditary angioedema. Would be the first in vivo CRISPR gene editing therapy approved anywhere, given as a single one-time infusion.
What happens after the Lonvo-z decision?
If approved, availability is not immediate: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. If the FDA issues a complete response letter, the application was not approved in its current form; CRLs are often about manufacturing or data presentation rather than efficacy, and sponsors frequently resubmit. This page updates with the outcome either way.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from Intellia press release. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.