Hemgenix
A single-dose intravenous gene therapy used to treat adults with severe Hemophilia B.
Where Etranacogene Dezaparvovec is approved
All regulators →Of the three regulators tracked here, the first to approve Etranacogene Dezaparvovec was the EU, on 20 Feb 2023. It is approved in 2 of the 3 regulators tracked here.
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Etranacogene Dezaparvovec patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Etranacogene Dezaparvovec, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- Hemgenix for treatment of haemophilia B: until 23 Feb 2033. Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 12 Oct 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
How Etranacogene Dezaparvovec works
All drug targets →- Coagulation factor IX exogenous protein Exogenous proteinTarget: F9 coagulation factor IXChEMBL only
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL5095451, CC BY-SA 3.0. The Guide to Pharmacology has no record of this medicine, so its targets rest on ChEMBL alone. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Etranacogene Dezaparvovec
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Etranacogene Dezaparvovec. 1 is current.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B NICE TA989 | Optimised (Innovative Medicines Fund) | 24 Jul 2024 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Etranacogene Dezaparvovec
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- EUEuropean Union: HemgenixEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
What Hemgenix is used for
HEMGENIX is indicated for treatment of adults with Hemophilia B (congenital Factor IX deficiency) 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 (congenital Factor IX deficiency) who: Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage, or Have repeated, serious spontaneous bleeding episodes.
How it works
12.1 Mechanism of Action HEMGENIX is an adeno-associated virus serotype 5 (AAV5) based gene therapy designed to deliver a copy of a gene encoding the Padua variant of human coagulation Factor IX (hFIX-Padua). Single intravenous infusion of HEMGENIX results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.
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How this page is built
The facts on this page are pulled directly from official U.S. FDA datasets — they are not written from memory. Each field below names the dataset it came from, so you can check it yourself.
- FDA Drug Label (openFDA) ↗ Indications, mechanism of action and pharmacologic class
- Drugs@FDA ↗ First FDA approval date and the company holding the application
- FDA Drug Shortage Database ↗ Current US supply / shortage status
Plain-English summaries and drug-class explainers are written and reviewed by Sreepriya Prasannan (MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany). Data is retrieved automatically from the sources above and cross-checked with AI-assisted verification (Anthropic's Claude) — brand and generic names are matched against the exact FDA product record so that a combination product or a different formulation cannot be mistaken for the drug on this page. An editor reviews the result before publication. We describe this in full in our editorial standards and corrections policy. The FDA data on this page was last retrieved on 6 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.