Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B (TA989)

Clinical guideline · Tier 1, UK official · UK · 2024-07-24

Current — checked with the publisher on 2026-09-02

https://www.nice.org.uk/guidance/ta989/chapter/1-Recommendation

What this platform has drawn from it — 2 claims

Etranacogene dezaparvovec holds a conditional marketing authorisation for the treatment of severe and moderately severe haemophilia B, a congenital factor IX deficiency, in adult patients without a history of factor IX inhibitors. Haemophilia B is an inherited bleeding disorder, not a disease of the liver. Chapter 2 Information about etranacogene dezaparvovec, section 2.1
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NICE recommends etranacogene dezaparvovec with managed access for treating moderately severe or severe haemophilia B in adults without anti-factor IX antibodies, and only if the conditions in the managed access agreement are followed. Chapter 1 Recommendation, section 1.1, first sentence · Chapter 1 Recommendation, section 1.1, second sentence
read on Gene therapy and the liver

How this record was checked, and what is doubtful about it

Held on this platform ONLY because the therapy is delivered to hepatocytes and its eligibility and safety turn on liver health. The indication is haemophilia B, a bleeding disorder. nice.org.uk 403s plain fetchers; reached through a browser session.

Verification: verified · last accessed 2026-09-02 · status checked against https://www.nice.org.uk/guidance/ta989