Also known as: sirolimus
Rapamycin (sirolimus) is an mTOR inhibitor immunosuppressant used clinically for prevention of renal transplant rejection and for lymphangioleiomyomatosis. Its label describes inhibition of T-lymphocyte activation/proliferation through FKBP-12 complex binding to mTOR. U.S. labeling indicates initial FDA approval in 1999.
FDA granted rolling review for Palvella's QTORIN rapamycin NDA. Also covered: global expedited approval pathways, accelerated approval scrutiny, and multi-regional CDx strategies.
Quoin Pharmaceuticals has secured Orphan Drug Designations for QRX003 in the U.S. and Europe for Netherton Syndrome, while advancing regulatory pathways in Japan and Saudi Arabia. The company reported clinical progress including expansion of pediatric studies and potential for a Priority Review Voucher worth up to $200 million.
Interim results from the ABC phase 1/2b trial show a calcineurin inhibitor-free GVHD prevention regimen combining posttransplant cyclophosphamide, bortezomib, and abatacept achieved low GVHD rates and favorable survival outcomes. The approach enables earlier introduction of posttransplant maintenance therapies while reducing toxicity associated with conventional immunosuppressants.
Researchers develop multiple strategies to enhance CAR T-cell therapy, including dual PGE2 receptor knockout for solid tumors and small-molecule switches using venetoclax and caffeine to control cellular activity and improve safety.
Palvella Therapeutics announced positive topline results from its Phase 3 SELVA study of QTORIN 3.9% rapamycin for microcystic lymphatic malformations, meeting all primary and secondary endpoints with strong tolerability. The company plans an NDA submission in H2 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07475546 |
Combination Gerotherapeutic Interventions for Healthspan Improvement |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT07092618 |
Effectiveness of Alternative Therapies in Maintaining Weight Loss Achieved by GLP-1 Medications Post-Cessation |
RECRUITING | PHASE2/PHASE3 |
| NCT07058974 |
A Study of Exercise and Pharmacologic Intervention on Systemic Inflammation |
RECRUITING | PHASE1 |
| NCT06743503 |
UB-VV400 in Combination With Rapamycin in Relapsed or Refractory B-cell Malignancies |
RECRUITING | PHASE1 |
| NCT06658093 |
RESTOR: PK/PD mTORi Inhibition in Older Adults |
RECRUITING | EARLY_PHASE1 |
| NCT06563817 |
The Safety and Efficacy of Rapamycin on Communicating Hydrocephalus Secondary to Intraventricular Hemorrhage |
RECRUITING | PHASE2 |
| NCT06550271 |
The Bioavailability of Compounded and Generic Rapamycin in Normative Aging Individuals |
COMPLETED | |
| NCT06528301 |
A Phase 1 Study of UB-VV111 With and Without Rapamycin in Relapsed/Refractory CD19+ B-cell Malignancies |
RECRUITING | PHASE1 |
| NCT06308445 |
Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis |
NOT_YET_RECRUITING | PHASE2 |
| NCT06257420 |
Low Dose Rapamycin in ME/CFS, Long-COVID, and Other Infection Associated Chronic Conditions |
ENROLLING_BY_INVITATION |
