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Drugs in development · updated 30 October 2026

Von Hippel-Lindau disease: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:892

As of 30 October 2026 there are 5 active clinical trials (recruiting or ongoing, phases I–III) on 5 drugs for von Hippel-Lindau disease. One is experimental, that is not yet approved in the United States.

5Active trials
1Experimental drugs
0Experimental, 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
HS-10516I1NCT06553339

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
BelzutifanWelireg®III4NCT07405164
CabozantinibResniben®, Cabometyx®III1NCT07405164
LenvatinibLenvatinib Mesilate Capsules, LevatinibIII1NCT07405164
NivolumabOpdivo, ONO-4538III1NCT07405164

Sorted by most advanced phase and number of active trials.

All 5 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: D006623 “von Hippel-Lindau Disease”. Prevalence: Orphanet.

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