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Drugs in development · updated 30 October 2026

Retinitis pigmentosa: drugs in development

Rare diseasePrevalence 1-5 / 10 000 according to Orphanet · ORPHA:791

As of 30 October 2026 there are 27 active clinical trials (recruiting or ongoing, phases I–III) on 25 drugs for retinitis pigmentosa. 20 are experimental, that is not yet approved in the United States.

27Active trials
20Experimental drugs
3Experimental, 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
RTx-015II–III2NCT07901751
N-acetylcysteine amideNACAIII1NCT07290530
NPI-001III1NCT07710196
ZVS203eI–II2NCT06952842
AAVB-081I–II1NCT06591793
BS01I–II1NCT04278131
DSP-3077I–II1NCT06891885
OCT-980I–II1NCT07408232
OCU400I–II1NCT05203939
OpCT-001I–II1NCT06789445
QR-1123I–II1NCT04123626
SPVN06I–II1NCT05748873
SPVN20I–II1NCT07807111
ZM-02I–II1NCT07282457
UGX202I2NCT07847723
CD34I1NCT01736059
IGT001I1NCT06936787
VG901I1NCT06291935
ZM-02-HEarly I1NCT06292650
ZM-02-LEarly I1NCT06292650

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
MinocyclineMinocyclin, Emrosi®III1NCT07082855
N-AcetylcysteineAcetylcysteine, AcetadoteIII1NCT05537220
Voretigene neparvovec-rzylLuxturna®III1NCT00999609
AdalimumabHadlima, Simlandi®I–II1NCT07348588
DisulfiramAntabuse®I1NCT06319872

Sorted by most advanced phase and number of active trials.

All 25 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: D012174 “Retinitis Pigmentosa”. Prevalence: Orphanet.

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