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Drugs in development · updated 30 October 2026

Hemophilia B: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:98879

As of 30 October 2026 there are 14 active clinical trials (recruiting or ongoing, phases I–III) on 10 drugs for hemophilia B. 7 are experimental, that is not yet approved in the United States.

14Active trials
7Experimental drugs
2Experimental, in phase III

Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
SR604III3NCT07876674
ANB-002III2NCT06700096
AskBio009I–II1NCT01687608
LNP1265I–II1NCT06379789
REGV131I–II1NCT06379789
ZS801I–II1NCT05641610
BBM-H901I1NCT05709288

Sorted by most advanced phase and number of active trials.

Approved drugs in new trials

Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.

DrugMost advanced phaseActive trialsReference trial
CSL222III2NCT07080905
MarstacimabPF-06741086, Hympavzi®III2NCT05145127
Fidanacogene elaparvovecBeqvez®, Beqvez (previously Durveqtix)®III1NCT03861273

Sorted by most advanced phase and number of active trials.

All 10 drugs, with the details

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Broader categories

Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D002836 “Hemophilia B”. Prevalence: Orphanet.

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