Section 3 of 6
Product information and disease description
66 evidence topics · 49 sources
Product description
Phase of product development
Regulatory submission: Rolling BLA initiated April 2026 and accepted September 2026
FDA acceptance, review designation, target action date, and advisory committee
“accepted the Biologics License Application (BLA)” (opens the source at this quote in a new tab)
“Priority Review” (opens the source at this quote in a new tab)
“PDUFA target action date of March 10, 2027” (opens the source at this quote in a new tab)
“not currently planning to hold an advisory committee” (opens the source at this quote in a new tab)
Launch: Anticipated in the first half of 2027
FDA Regenerative Medicine Advanced Therapy designation
Additional international regulatory designations: European Union
Additional international regulatory designations: United Kingdom
Additional international regulatory designations: European Medicines Agency
Reviews and editorial publications: kallikrein inhibitors in preclinical and early phase studies
Product information
Generic, brand name and therapeutic class of product
Dosage forms and strengths
Material safety data sheet
No evidence found.
Average sales price and wholesale acquisition cost
Average sales price and wholesale acquisition cost
Not applicable.
American hospital formulary service (AHFS), or other drug classification
Therapeutic modality
Indication
Investigational population in the phase 3 trial supporting the BLA
Pharmacology
Mechanism of action
Ownership, therapeutic class, and proposed mechanism
Hepatic targeting for novel prophylactic therapies: review
Pharmacodynamics
Phase 3: plasma kallikrein time course
Preclinical studies: editing durability after partial hepatectomy in huKLKB1 mice
Preclinical studies: KLKB1 gene knockout in nonhuman primates
Pharmacokinetics
Pharmacokinetics: lipid nanoparticle component exposure and clearance
Contraindications/Warnings/Precautions/Adverse effects
Warnings and precautions
Not applicable.
Special populations
No evidence found.
Drug/Drug, drug/disease interactions
Effects of other drugs on Lonvoguran ziclumeran
No evidence found.
Effects of Lonvoguran ziclumeran on other drugs
No evidence found.
Dosing and administration
Dosage
Administration
Route and setting of administration
Infusion duration in the phase 1/2 study
Access and distribution
Planned distribution and treatment-center identification
Co-prescribed/Concomitant therapies
HAELO: withdrawal of long-term prophylaxis
Effect of Lonvoguran ziclumeran on quality measures
No evidence found.
Product comparison
Place of product in therapy
Disease description
Definition and etiology
Epidemiology
Incidence of Hereditary angioedema
Prevalence of Hereditary angioedema
Prevalence and incidence: systematic literature review
Pooled diagnosed annual prevalence: combined HAE types and 95% confidence interval
Natural history, survival, and mortality
Pathophysiology
Diagnosis
Systematic literature review of HAE disease burden: diagnostic delay
Clinical presentation - signs and symptoms
Gastrointestinal symptoms: intestinal attacks
Airway symptoms: obstruction risk
Long-term morbidity
Consequences of abdominal attacks
Burden of Hereditary angioedema
Humanistic burden and health-related quality of life
Burden of disease in adults: multinational survey
Multinational adult survey: treatment exposure and quality-of-life outcomes
| Measure | Reported result |
|---|---|
| Participants included | “260” |
| Participants using any long-term prophylaxis | “153 (58.8%)” |
| First-line prophylaxis among prophylaxis users, per study classification | “56/153 (36.6%)” |
| AE-QoL total score, mean ± SD | “42.9 ± 23.2” |
| Participants with AE-QoL total score ≥39 | “137 of 260 (52.7%)” |
| Moderate to severe anxiety by HADS | “69 (26.5%)” |
| Moderate to severe depression by HADS | “27 (10.4%)” |
Patient preferences for long-term prophylaxis: discrete-choice experiment
Economic burden and healthcare resource utilization
Economic impact of Hereditary angioedema on families
Income loss from work missed because of attacks
Economic impact of diagnostic testing
Genetic testing: cost and limited routine necessity
Approaches to treatment
Current treatment options and standard of care
C1 esterase inhibitors
International/Canadian guideline: first-line long-term prophylaxis options
Plasma kallikrein inhibitors
Bradykinin b2 receptor antagonists
Factor XIIa inhibitors
Garadacimab: mechanism and maintenance dosing
Prekallikrein-directed antisense oligonucleotides
Donidalorsen: prophylaxis and dosing interval
Attenuated androgens
Second-line long-term prophylaxis
Antifibrinolytics
Tranexamic acid: reported prophylactic responses in HAE-FXII, HAE-PLG, and HAE with normal C1 inhibitor of unknown cause
Androgens and antifibrinolytics: restriction on first-line use
Plasma replacement
Fresh frozen plasma: fallback for on-demand treatment
Progestins
Progestin prophylaxis: variant-specific observational evidence
| Feature | Guideline description |
|---|---|
| Subtype with reported responses | “HAE-FXII” |
| Reported response | “Favourable responses” |
| Evidence for other variants | “limited evidence for other HAE-nC1INH variants.” |
Supportive care and airway management
Supportive care when effective on-demand treatment is not available
Limitations of current therapies
Summary
The multinational adult survey reported a mean of 11.5 attacks over six months, but only 56 of 153 participants using prophylaxis reported a first-line option under the study classification. These findings should not be interpreted as a failure rate for current first-line prophylaxis or as a controlled comparison of drugs. A claims study reported annualized expenditure above $200,000 for on-demand prescriptions, hospitalizations, and emergency department visits even after subcutaneous prophylaxis began. The Cochrane review identified limitations in drawing conclusions about relative efficacy because head-to-head trials were lacking.
Long-term prevention of attacks: Cochrane review
Place in treatment, anticipated use, and care setting
Summary
The trial supporting the accepted BLA permitted enrollment of adults and adolescents aged 16 years or older with type 1 or type 2 HAE. The investigational regimen is a single 50-mg intravenous infusion, with outpatient administration anticipated. HAELO required discontinuation of long-term prophylaxis before dosing, so its placebo-controlled findings should not be interpreted as an active-comparator trial or as evidence for routine combination with ongoing prophylaxis. The investigational regimen does not replace the guideline requirement for rapid access to on-demand treatment.
Heterogeneity of treatment effect
HAELO: sponsor-reported subgroup findings
Care management intervention strategies
US HAEA Medical Advisory Board guidelines: on-demand medication access
Other product development or post-marketing obligations required by the FDA
Not applicable.
Ongoing post-approval monitoring
Not applicable.