Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Pulmonary Arterial Hypertension (PAH) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Pulmonary Arterial Hypertension (PAH). ClinicalTrials.gov lists 1 registered trial linking Sirolimus to Pulmonary Arterial Hypertension (PAH): 1 has completed. The most advanced is Phase 1 (NCT02587325). It plans or enrolled 15 participants. Registered activity dates to 2017. No published literature item is linked to Sirolimus and Pulmonary Arterial Hypertension (PAH) in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Pulmonary Arterial Hypertension ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Without treatment, median survival from diagnosis 2.8 years (pre-modern-therapy era); with current therapy 5-year survival ~50-60%; spontaneous remission essentially unknown.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 23.0 (trials 3.0, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 1 total: 0 recruiting, 0 active / not yet recruiting, 1 completed, 0 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ABCC8 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT02587325 | Phase 1/1b Study With Nab-sirolimus for Patients With Severe Pulmonary Arterial Hypertension 2017 | completed | Phase 1 | 15 |
Published literature
No publication is linked to this pair yet.
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target ABCC8. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Pulmonary Arterial Hypertension (PAH)?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Pulmonary Arterial Hypertension (PAH) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Pulmonary Arterial Hypertension (PAH). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Pulmonary Arterial Hypertension (PAH)?
ClinicalTrials.gov lists 1 registered trial linking Sirolimus to Pulmonary Arterial Hypertension (PAH): 1 has completed. The most advanced is Phase 1 (NCT02587325). It plans or enrolled 15 participants. Registered activity dates to 2017.
What does the evidence show for Sirolimus in Pulmonary Arterial Hypertension (PAH)?
Sirolimus has 1 completed registered trial for Pulmonary Arterial Hypertension (PAH) but no linked publication, which usually means results are unpublished, pending, or not yet matched to this record. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target ABCC8. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.