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Drugs in development · updated 30 October 2026
Rare disease: drugs in development
As of 30 October 2026 there are 14 active clinical trials (recruiting or ongoing, phases I–III) on 12 drugs for rare disease. 8 are experimental, that is not yet approved in the United States.
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.
Sign in free →Experimental drugs
Not yet approved by the US FDA for any indication.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| RAPA-501 | II–III | 1 | NCT04220190 |
| S-606001 | II | 3 | NCT07750990 |
| CTH120 | II | 1 | NCT07654114 |
| KK8123 | I–II | 1 | NCT06525636 |
| PBGM01 | I–II | 1 | NCT04713475 |
| SPK-10001 | I–II | 1 | NCT06826612 |
| Votoplam | II | 1 | NCT06254482 |
| SGT-212 | I | 1 | NCT07180355 |
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.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| CannabidiolEpidiolex, Epidyolex® | III | 2 | NCT07668856 |
| InebilizumabUplizna® | II | 1 | NCT04372615 |
| IpilimumabYERVOY, ipililumab IV | II | 1 | NCT02834013 |
| NivolumabOpdivo, ONO-4538 | II | 1 | NCT02834013 |
Sorted by most advanced phase and number of active trials.
All 12 drugs, with the details
In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.
Open in Phuturdex →Broader categories
- Nervous System Diseases 39 drugs
- Central nervous system disease 38 drugs
- Brain Diseases 23 drugs
- Genetic disease 33 drugs
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Nutritional and metabolic diseases 5 drugs
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: D035583 “Rare Diseases”.