Hemophilia B, severe

Severe hemophilia B / Factor IX deficiency, severe / Hemophilia B due to F9 deficiency

8.4
Overall
Confidence: 82%
Composite of urgency, severity, and feasibility — higher score indicates greater research priority
MONDO:MONDO:0010600F9 ↗

Global prevalence of all hemophilia B is ~3.8/100,000 males and severe hemophilia B ~1.1/100,000 males, implying ~289,000 severe cases worldwide; disease is concentrated in countries with established registries and hemophilia treatment centers.

Approved drugs
FDA approved

Fibrinogen + PCC

University Health Network, Toronto

Fibrinogen exogenous protein

Other
FDA approved

Frozen Plasma

University Health Network, Toronto

Mechanism not available yet.

Other
Drugs in Pipeline
31
2 approved · 13 Phase 3 · 9 Phase 2 · 6 Phase 1
→
Clinical Trials
50
3 recruiting · 1 active · 36 completed · 10 other
→
Evidence Sources (26)
webHuman Gene Therapy for Hemophilia – Guidance for Industry0.96↗paperGene Therapy with Etranacogene Dezaparvovec for Hemophilia B0.95↗webReport on the Annual Global Survey 20240.95↗webWFH Guidelines for the Management of Hemophilia, 3rd edition (2020)0.95↗paperCOAGULATION FACTOR IX; F90.93↗

Last updated: March 26, 2026

Data sources: ClinVar 2026-03 · gnomAD v4.1 · ClinicalTrials.gov API v2 · MONDO:MONDO:0010600