Drugs / Rapamycin
last change Sep 2026 re-read 3 minutes ago

Rapamycin

Small molecule targets IL2, MTOR via inhibition

Developed for
type 1 diabetes mellitus · tuberous sclerosis · acute myeloid leukemia · breast cancer · cancer of unknown primary site · capillary malformation · classic familial adenomatous polyposis · premature aging syndrome
+81 more · acute lymphoblastic leukemia · chronic myeloid leukemia · neoplasm · pachyonychia congenita · prostate cancer · urinary bladder carcinoma · vascular malformation · vascular neoplasm · activated PI3K-delta syndrome · acute leukemia of ambiguous lineage · autosomal dominant polycystic kidney disease · benign vascular tumor · Birt-Hogg-Dube syndrome · blue rubber bleb nevus · Burkitt lymphoma · cancer · cancer or benign tumor · central nervous system neoplasm · cervical squamous cell carcinoma · childhood malignant neoplasm · Crohn disease · disease of orbital part of eye adnexa · endometrial carcinoma · female breast carcinoma · graft versus host disease · head and neck carcinoma · heart failure · inclusion body myositis · infertility disorder · keloid · kidney angiomyolipoma · kidney disorder · kidney failure · large B-cell lymphoma · leiomyoma · liver cancer · low grade glioma · lymphatic malformation · malignant pancreatic neoplasm · metastatic malignant neoplasm · metastatic neoplasm · microcystic lymphatic malformation · neuroblastoma · neurofibromatosis · non-small cell lung carcinoma · normal pressure hydrocephalus · osteosarcoma · ovarian carcinoma · Peutz-Jeghers syndrome · pineoblastoma · plasma cell myeloma · precursor lymphoblastic lymphoma/leukemia · premature menopause · prostate carcinoma · rectal cancer · renal cell carcinoma · Sjogren syndrome · skin atrophy · skin cancer · soft tissue sarcoma · Sturge-Weber syndrome · systemic lupus erythematosus · systemic sclerosis · transplant rejection · uterine adnexa cancer · acute graft versus host disease · acute respiratory distress syndrome · age-related macular degeneration · alcohol dependence · Alzheimer disease · amyotrophic lateral sclerosis · choroidal neovascularization · chronic graft versus host disease · cognitive impairment with or without cerebellar ataxia · COVID-19 · hepatocellular carcinoma · non-invasive bladder urothelial carcinoma · pancreatic adenocarcinoma · post-traumatic stress disorder · urinary bladder cancer · vitiligo
Investigated by
National Cancer Institute (NCI) · Pfizer · University of Chicago · The University of Texas Health Science Center at San Antonio · Yale University · Abramson Cancer Center at Penn Medicine
+97 more · AgelessRx · Mayo Clinic · Palvella Therapeutics, Inc. · Peking Union Medical College Hospital · Air Force General Hospital of the PLA · Beckman Laser Institute University of California Irvine · Biodexa Pharmaceuticals · Cancer Insight, LLC · Genentech, Inc. · Italian Diabetes Foundation · National Institute on Aging (NIA) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · University Hospital, Toulouse · University of California, Irvine · University of California, Los Angeles · University of Regensburg · Wyeth is now a wholly owned subsidiary of Pfizer · AFT Pharmaceuticals, Ltd. · Astellas Pharma Inc · Bayer · Beijing Tiantan Hospital · Beijing Tongren Hospital · Broto, Javier Martín, M.D. · Cancer Institute and Hospital, Chinese Academy of Medical Sciences · Cheloo Hospital (Qingdao), Shandong University · Children's Hospital of Fudan University · Children's Hospital of Philadelphia · Children's National Research Institute · Chinese Academy of Medical Sciences · Clinica Universidad de Navarra, Universidad de Navarra · Columbia University · Dana-Farber Cancer Institute · Danish Diabetes Academy · Dermatology Specialties Limited Partnership · Drexel University · FDA Office of Orphan Products Development · First Affiliated Hospital Xi'an Jiaotong University · Fondazione IRCCS Policlinico San Matteo di Pavia · French Innovative Leukemia Organisation · French National Institute of Health and Medical Research · Gustave Roussy, Cancer Campus, Grand Paris · Heidelberg University · Hospices Civils de Lyon · IRCCS San Raffaele · Icahn School of Medicine at Mount Sinai · Immune Tolerance Network (ITN) · Jiangsu Province Hospital of Traditional Chinese Medicine · Jonsson Comprehensive Cancer Center · Juvenile Diabetes Research Foundation · Katarzyna Kotulska · LuZhe Sun · Maastricht Radiation Oncology · Maastricht University Medical Center · Maastricht University Medical Center · Mehdi Yousefi · Ministry of Education, Universities and Research, Italy · Ministry of Health, France · Myrovlytis Trust · Nanjing First Hospital, Nanjing Medical University · Nanjing IASO Biotechnology Co., Ltd. · National Cancer Centre, Singapore · National Institute of Allergy and Infectious Diseases (NIAID) · National Institutes of Health (NIH) · National Taiwan University Hospital · Ospedale San Raffaele · Peking Union Medical College · Peking University People's Hospital · People's Hospital of Xinjiang Uygur Autonomous Region · Piemonti Lorenzo · Rapamycin Holdings Inc. · Rapamycin Holdings, Inc. dba Emtora Biosciences · Roger Packer · Sociedad Andaluza de Trasplantes de Organos y Tejidos · Society for Pediatric Dermatology · all 103 on the full record

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PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 08 trials
Phase 0 NCT06658093 Mar 2026 → Jul 2028 expected — The University of Texas Health Science Center at San Antonio Recruiting No outcome recorded
Phase 0 NCT07354620 Feb 2025 → Feb 2026 overdue premature aging syndrome The Christ Hospital Active not recruiting No outcome recorded
Phase 0 NCT05532072 Sep 2022 → Sep 2023 disease of orbital part of eye adnexa First Affiliated Hospital Xi'an Jiaotong University Unknown No outcome recorded
Phase 0 NCT04049552 Jan → Aug 2020 keloid The University of Texas Health Science Center at San Antonio Completed No outcome recorded
Phase 0 NCT02646319 Jan 2016 → Sep 2017 cervical squamous cell carcinoma, endometrial carcinoma, female breast carcinoma, head and neck carcinoma +5 Mayo Clinic Completed No outcome recorded
Phase 0 NCT01827618 Apr 2012 → Apr 2014 urinary bladder carcinoma The University of Texas Health Science Center at San Antonio Completed No outcome recorded
Phase 0 NCT01396200 Jun 2011 → Oct 2012 plasma cell myeloma Abramson Cancer Center at Penn Medicine Completed No outcome recorded
Phase 0 NCT00583063 Oct 2007 → Apr 2008 cancer University of Chicago Completed No outcome recorded
Phase 123 trials
Phase 1 NCT07058974 Aug 2025 → Jul 2026 overdue — Icahn School of Medicine at Mount Sinai Recruiting No outcome recorded
Phase 1 NCT06743503 Apr 2025 → May 2028 expected large B-cell lymphoma Nanjing IASO Biotechnology Co., Ltd. Recruiting No outcome recorded
Phase 1 NCT06528301 Mar 2025 → Mar 2029 expected B-cell chronic lymphocytic leukemia, diffuse large B-cell lymphoma Umoja Biopharma Recruiting No outcome recorded
Phase 1 NCT04994561 Jun 2022 → Dec 2023 — Vitality in Aging Research Group, Inc. Withdrawn No outcome recorded
Phase 1 NCT04996719 May 2022 → Aug 2023 heart failure Mayo Clinic Completed No outcome recorded
Phase 1 NCT03618355 Aug 2018 → Dec 2019 prostate cancer Rapamycin Holdings, Inc. dba Emtora Biosciences Completed No outcome recorded
Phase 1 NCT01649960 Aug 2012 → Dec 2015 premature aging syndrome Mayo Clinic Completed No outcome recorded
Phase 1 NCT01282697 Apr 2011 → Feb 2013 childhood malignant neoplasm University Hospital, Strasbourg, France Completed No outcome recorded
Phase 1 NCT00861874 Jan 2010 → Sep 2012 acute myeloid leukemia University of Rochester Completed No outcome recorded
Phase 1 NCT01031901 Dec 2009 → Feb 2011 neurofibroma, neurofibromatosis, tuberous sclerosis, vascular neoplasm The University of Texas Health Science Center, Houston Completed No outcome recorded
Phase 1/210 trials
Phase 1/2 NCT05605665 Dec 2022 → Dec 2023 Sjogren syndrome Peking University People's Hospital Completed No outcome recorded
Phase 1/2 NCT03383380 Dec 2017 → Nov 2022 activated PI3K-delta syndrome Children's Hospital of Fudan University Completed No outcome recorded
Phase 1/2 NCT03103893 Sep → Nov 2017 skin atrophy Drexel University Completed No outcome recorded
Phase 1/2 NCT01684449 Jan 2010 → May 2013 soft tissue sarcoma Spanish Group for Research in Sarcomas Completed No outcome recorded
Phase 1/2 NCT00623012 Feb 2008 → Aug 2013 graft versus host disease Yale University Terminated No outcome recorded Stop: Enrollment
Phase 1/2 NCT00776373 Jan 2007 → Dec 2008 Burkitt lymphoma, chronic myeloid leukemia, precursor lymphoblastic lymphoma/leukemia Abramson Cancer Center at Penn Medicine Terminated No outcome recorded
Phase 1/2 NCT00409994 Sep 2006 → Aug 2018 rectal cancer Maastricht Radiation Oncology Completed No outcome recorded
Phase 1/2 NCT00311623 Aug 2006 → Jan 2008 prostate cancer Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Completed No outcome recorded
Phase 1/2 NCT00241189 Aug 2002 → Jun 2006 systemic sclerosis University of California, Los Angeles Completed No outcome recorded
Phase 1/2 NCT00047073 Jul 2002 → Jun 2005 central nervous system neoplasm Jonsson Comprehensive Cancer Center Completed No outcome recorded
Phase 225 trials · 2 met primary · 1 missed
Phase 2 NCT06308445 Sep 2026 → Sep 2030 expected classic familial adenomatous polyposis University Hospital, Toulouse Not yet recruiting No outcome recorded
Phase 2 NCT07436611 Aug 2026 → Oct 2027 expected capillary malformation AFT Pharmaceuticals, Ltd. Not yet recruiting No outcome recorded
Phase 2 NCT06563817 Aug 2024 → May 2025 overdue intracerebral hemorrhage, normal pressure hydrocephalus Beijing Tiantan Hospital Recruiting No outcome recorded
Phase 2 NCT05836025 Jun 2023 → Oct 2025 overdue premature menopause Columbia University Active not recruiting No outcome recorded
Phase 2 NCT04742777 Feb 2022 → Sep 2025 — The University of Texas Health Science Center at San Antonio Completed No outcome recorded
Phase 2 NCT04230499 Jan 2021 → Mar 2025 overdue classic familial adenomatous polyposis Rapamycin Holdings Inc. Active not recruiting Met primary
Phase 2 NCT04488601 Jan 2020 → Dec 2023 premature aging syndrome AgelessRx Completed No outcome recorded
Phase 2 NCT03243019 Jun 2018 → Feb 2027 expected lymphatic malformation University Hospital, Lille Recruiting No outcome recorded
Phase 2 NCT03161340 Jul 2017 → May 2018 infertility disorder Mehdi Yousefi Completed No outcome recorded
Phase 2 NCT02642094 Jul 2016 → Mar 2022 breast cancer LuZhe Sun Terminated Mixed
Phase 2/37 trials · 2 missed
Phase 2/3 NCT07092618 Dec 2024 → Aug 2025 overdue — AgelessRx Recruiting No outcome recorded
Phase 2/3 NCT04987463 May 2021 → Mar 2026 tuberous sclerosis Katarzyna Kotulska Unknown No outcome recorded
Phase 2/3 NCT03826628 Jul 2019 → Sep 2022 tuberous sclerosis, vascular neoplasm Dermatology Specialties Limited Partnership Completed No outcome recorded
Phase 2/3 NCT03920228 Apr 2019 → Sep 2020 pachyonychia congenita Palvella Therapeutics, Inc. Completed Missed primary
Phase 2/3 NCT04598204 Dec 2017 → Dec 2022 vascular malformation Zhujiang Hospital Unknown No outcome recorded
Phase 2/3 NCT02481453 Jul 2015 → Jan 2018 inclusion body myositis French National Institute of Health and Medical Research Completed Missed primary
Phase 2/3 NCT00920309 Jun 2009 → Jul 2010 autosomal dominant polycystic kidney disease Yale University Terminated No outcome recorded
Phase 38 trials · 2 met primary · 1 missed
Phase 3 NCT07475546 Jul 2025 → Apr 2026 overdue — AgelessRx Active not recruiting No outcome recorded
Phase 3 NCT06239480 Jul 2024 → Jan 2026 overdue microcystic lymphatic malformation Palvella Therapeutics, Inc. Active not recruiting Met primary
Phase 3 NCT05643872 Nov 2022 → Oct 2023 overdue pachyonychia congenita Palvella Therapeutics, Inc. Recruiting No outcome recorded
Phase 3 NCT04736589 Feb 2021 → Feb 2024 overdue breast cancer Peking Union Medical College Not yet recruiting No outcome recorded
Phase 3 NCT03140449 Sep 2013 → Nov 2016 benign vascular tumor National Taiwan University Hospital Completed No outcome recorded
Phase 3 NCT00928798 Jan 2010 → Feb 2011 Birt-Hogg-Dube syndrome Maastricht University Medical Center Completed Missed primary
Phase 3 NCT00133887 Apr 2004 → Mar 2011 skin cancer Hospices Civils de Lyon Completed Met primary
Phase 3 NCT01060605 Oct 2001 → Dec 2005 type 1 diabetes mellitus IRCCS San Raffaele Completed No outcome recorded
Phase 45 trials
Phase 4 NCT03781050 Sep 2018 → Jan 2022 Peutz-Jeghers syndrome Peking Union Medical College Hospital Unknown No outcome recorded
Phase 4 NCT03767660 Jul 2018 → Jan 2022 blue rubber bleb nevus, vascular malformation Peking Union Medical College Hospital Unknown No outcome recorded
Phase 4 NCT03500367 Jan 2018 → Jun 2022 leiomyoma Peking Union Medical College Hospital Unknown No outcome recorded
Phase 4 NCT00123331 Oct 2003 → Apr 2005 kidney failure Heidelberg University Completed No outcome recorded
Phase 4 NCT00290069 — kidney disorder, transplant rejection Sociedad Andaluza de Trasplantes de Organos y Tejidos Unknown No outcome recorded
Phase not applicable3 trials
— NCT05233722 Feb 2022 → Jun 2029 expected — University of Copenhagen Recruiting No outcome recorded
— NCT02551718 Sep 2015 → Jun 2020 acute leukemia of ambiguous lineage, acute lymphoblastic leukemia University of Washington Completed No outcome recorded
— NCT02675153 Apr 2015 → Oct 2020 Crohn disease The Second Hospital of Nanjing Medical University Unknown No outcome recorded
Phase not stated1 trial
— NCT07689942 — classic familial adenomatous polyposis Biodexa Pharmaceuticals Available No outcome recorded
Comparator or background therapy25 trials
Phase 0 NCT04608448 Comparator Apr 2021 → Apr 2022 — The University of Texas Health Science Center at San Antonio Completed No outcome recorded
Phase 1 NCT05105152 Background Nov 2021 → Feb 2028 expected acute myeloid leukemia Seattle Children's Hospital Recruiting No outcome recorded
Phase 1 NCT03732248 Comparator Jul 2018 → Dec 2019 alcohol dependence Medical University of South Carolina Completed No outcome recorded
Phase 1 NCT02240407 Background Oct 2017 → Aug 2021 glycogen storage disease II University of Florida Completed No outcome recorded
Phase 1 NCT02048384 Comparator Jun 2014 → Feb 2018 pancreatic adenocarcinoma Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Completed No outcome recorded

Press releases naming this drug 24 releases

DateIssuerRelease
2026-07-27 CSPC ZhongQi Pharmaceutical Technology Co., Ltd. VOLUNTARY ANNOUNCEMENT - NEW DRUG APPLICATION FOR SIROLIMUS FOR INJECTION (ALBUMIN-BOUND) ACCEPTED BY THE NMPA irasia.com ↗
2026-07-16 CSPC ZhongQi Pharmaceutical Technology Co., Ltd. VOLUNTARY ANNOUNCEMENT - PHASE II CLINICAL TRIAL OF SIROLIMUS FOR INJECTION (ALBUMIN-BOUND) FOR THE TREATMENT OF EPITHELIOID HAEMANGIOENDOTHELIOMA OFFICIALLY INITIATED AT THE FIRST CENTRE IN CHINA irasia.com ↗
2026-03-23 Biodexa Pharmaceuticals Biodexa Launches Global Early Access Program for eRapa for FAP Patients Through Strategic Partnership with Tanner Pharma Group biodexapharma.com ↗
2025-05-22 Biodexa Pharmaceuticals Biodexa Announces Award of Additional $3.0M Grant from CPRIT to Support Registrational eRapa Phase 3 Program in FAP Brings Total CPRIT Grant Funding for eRapa Phase 3 Program to $20.0M biodexapharma.com ↗
2025-05-12 Biodexa Pharmaceuticals Biodexa Receives Orphan Drug Designation in Europe for eRapa in FAP biodexapharma.com ↗
2025-03-10 Biodexa Pharmaceuticals Biodexa Announces Successful Outcome of a Type C Meeting with FDA Regarding the Phase 3 Program for eRapa in FAP biodexapharma.com ↗
2025-03-06 Biodexa Pharmaceuticals Biodexa Announces Appointment of Precision for Medicine LLC as CRO for European Component of Phase 3 Study of eRapa in FAP biodexapharma.com ↗
2025-02-28 CSPC ZhongQi Pharmaceutical Technology Co., Ltd. VOLUNTARY ANNOUNCEMENT - SIROLIMUS FOR INJECTION (ALBUMIN-BOUND) WAS GRANTED BREAKTHROUGH THERAPY DESIGNATION IN CHINA irasia.com ↗
2025-02-24 Biodexa Pharmaceuticals Biodexa Announces Allowance of U.S. Patent Covering Oral Rapamycin Nanoparticle Preparations (“eRapa”) and Use biodexapharma.com ↗
2025-02-10 Biodexa Pharmaceuticals Biodexa Receives US FDA Fast Track Designation for eRapa in Familial Adenomatous Polyposis biodexapharma.com ↗
2024-06-24 Biodexa Pharmaceuticals Biodexa Announces Positive Phase 2 Clinical Trial Results of eRapa™ at 12 months in Familial Adenomatous Polyposis (FAP) biodexapharma.com ↗
2024-06-10 Biodexa Pharmaceuticals Twelve month Phase 2 Clinical Trial Results of eRapa™ in Familial Adenomatous Polyposis (FAP) to be Presented at Prestigious Biennial InSIGHT 2024 Meeting in Barcelona biodexapharma.com ↗

Evidence & citations 13 cited values

Every value below carries the sentence it was read from. 117 sources stand behind the page.

FieldValueCited text
Known as rapamycin “The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "PTX-022".” NCT05643872 ↗
114

“This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.” NCT03920228 ↗

“The purpose of this research is to see if the drug rapamycin will improve the heart's ability to pump by improving your oxygen consumption.” NCT04996719 ↗

“The experimental group received rapamycin 2 mg/day orally for 24 weeks on the basis of methylprednisolone pulse therapy” NCT05532072 ↗

“Infusion with SC-DARIC33 followed by intermittent oral rapamycin administration” NCT05105152 ↗

“Oral rapamycin is given as a monotherapy at a initial dose of 0.8mg/m2” NCT04598204 ↗

“pre-Tx rapamycin treatment (0.1 mg/kg/day) for at least 30 days” NCT01346085 ↗

“Rapamycin p.o.: 4 mg/d, (target 8-15 ng/ml) (starting day -7)” NCT03963024 ↗

“subjects will take a once weekly 6 mg dose of rapamycin” NCT04994561 ↗

“0.5mg eRapa (encapsulated rapamycin) oral capsules” NCT04375813 ↗

“Pralatrexate Rapamycin Regorafenib” NCT02551718 ↗

Show 104 more sources

Known as (RAPA, RapamuneR) (sirolimus) “(RAPA, RapamuneR) (sirolimus)” NCT00795886 ↗
Known as Encapsulated Rapamycin “The eRapa (encapsulated rapamycin) drug product consists of sub-micron rapamycin particles incorporated into poly(methyl methacrylate) polymer (Eudragit® L 100 / S 100).” NCT03618355 ↗
1

“Encapsulated Rapamycin (eRapa)” NCT04230499 ↗

Known as eRapa “The eRapa (encapsulated rapamycin) drug product consists of sub-micron rapamycin particles incorporated into poly(methyl methacrylate) polymer (Eudragit® L 100 / S 100).” NCT03618355 ↗
1

“0.5mg eRapa (encapsulated rapamycin) oral capsules” NCT04375813 ↗

Known as PTX-022 “The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "PTX-022".” NCT05643872 ↗
1

“This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.” NCT03920228 ↗

Known as QTORIN “A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of...” NCT05180708 ↗
1

“QTORIN 3.9% Rapamycin Anhydrous Gel” NCT06239480 ↗

Known as RAPA “Inhibition of the mTOR pathway by rapamycin (RAPA), an immunosuppressive drug used as adjunct therapy in preventing solid organ allograft rejection, enhances longevity in mice.” NCT02874924 ↗
4

“rapamycin (RAPA), a key component of the immunosuppressive regimen in clinical islet transplantation” PMID 21084848 ↗ Jul 2009

“daily 0.5-1.0 ml applications of 8% RAPA (Rapamycin) in a petrolatum vehicle” NCT04049552 ↗

“Rapamycin (RAPA)” NCT00803010 ↗

“Sirolimus, RAPA” NCT04629495 ↗

Known as Rapamune “Rapamune® (rapamycin)” NCT02505893 ↗
Known as sirolimus “The purpose of this study is to evaluate the side effects of sirolimus (rapamycin) given in combination with chemotherapy (Mitoxantrone + Etoposide + Cytarabine (MEC)) on high...” NCT00780104 ↗
9

“Rapamycin (sirolimus) is a drug that targets the mTOR (mammalian target of rapamycin) protein involved in the PI3K-Akt signalling pathway downstream of PI3K.” NCT06308445 ↗

“This new formulation, encapsulated rapamycin (sirolimus), provides a more predictable bioavailability of this drug than \[the other formulation\].” NCT03618355 ↗

“Subject will be randomized to take Sirolimus (Rapamycin) 0.5mg once daily for 2 years or until disease recurrence” NCT03298958 ↗

“Evaluate the tolerability and efficacy of sirolimus (rapamycin) in combination with low-dose aracytin” NCT00235560 ↗

“test two of these drugs, Rapamycin (Sirolimus) and Everolimus (Afinitor), in healthy older adults” NCT06658093 ↗

“Rapamycin (Sirolimus) Treatment for Amyotrophic Lateral Sclerosis” NCT03359538 ↗

“plus 1 mg of sirolimus/rapamycin” NCT01031901 ↗

“Rapamycin (sirolimus)” NCT00375245 ↗

“Sirolimus, RAPA” NCT04629495 ↗

Action Inhibit “the mTOR inhibitor, sirolimus” PMID 21915260 ↗ Sep 2011
Modality Small molecule “It is a macrocyclic lactone” NCT00795886 ↗
Route Oral “2 mg by mouth daily” NCT00126672 ↗
Target MTOR “the mTOR inhibitor, sirolimus” PMID 21915260 ↗ Sep 2011