Hemophilia B Treatments

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Brand Name

Hemgenix

Generic Name
Etranacogene Dezaparvovec
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FDA approval date: November 22, 2022
Form: Kit

What is Hemgenix (Etranacogene Dezaparvovec)?

HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for treatment of adults with Hemophilia B who: Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage, or Have repeated, serious spontaneous bleeding episodes. HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B who: Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage, or Have repeated, serious spontaneous bleeding episodes.

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Related Clinical Trials

Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
Enrollment Status: Recruiting
Publish Date: April 16, 2026
Intervention Type: Genetic
Study Phase: Phase 3

Summary: This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.

Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies
Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies
Enrollment Status: Recruiting
Publish Date: March 17, 2026
Intervention Type: Genetic
Study Phase: Phase 3

Summary: The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.

An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
Enrollment Status: Recruiting
Publish Date: February 19, 2026
Intervention Type: Genetic, Biological

Summary: This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.

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Related Latest Advances

Signals of Toxicity Associated With Gene Therapy: The Case of Etranacogene Dezaparvovec in the Treatment of Hemophilia B.
Signals of Toxicity Associated With Gene Therapy: The Case of Etranacogene Dezaparvovec in the Treatment of Hemophilia B.
Journal: The Journal of pharmacy technology : jPT : official publication of the Association of Pharmacy Technicians
Published: April 06, 2026
CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.
CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.
Journal: Molecular therapy. Nucleic acids
Published: January 07, 2026
Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.
Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.
Journal: The New England journal of medicine
Published: December 08, 2025
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