> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/asset/sirolimus-08da04e4-ea95-4587-95c4-83d583e56570 > Record changed: 2026-10-13 # Sirolimus Highest phase reached Approved Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) Drug type Small molecule Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) First approval year 1999 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) ## Development status **First approval year** 1999 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) **Listing at source** Listed **Highest phase reached** Approved Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) ## Names and chemistry **Drug type** Small molecule Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) **Drug name** Sirolimus Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) **Molecular weight** 914.19 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) **Structure (SMILES)** CO\[C@H\]1C\[C@@H\]2CC\[C@@H\](C)\[C@@\](O)(O2)C(=O)C(=O)N2CCCC\[C@H\]2C(=O)O\[C@H\](\[C@H\](C)C\[C@@H\]2CC\[C@@H\](O)\[C@H\](OC)C2)CC(=O)\[C@H\](C)/C=C(\\C)\[C@@H\](O)\[C@@H\](OC)C(=O)\[C@H\](C)C\[C@H\](C)/C=C/C=C/C=C/1C Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) ### Brand names (10) - fyarro - Fyarro - hyftor - Hyftor - rapamune - Rapamune - sirolimus - Sirolimus - sirolimus component of nab rapamycin rapamycin - Sirolimus component of nab-rapamycin rapamycin ### Other names (138) - (-)-rapamycin - (-)-RAPAMYCIN - \[3S-\[3R\*\[S\* (1R\*,3S\*,4S\*)\],6S\*,7E,9S\*,10S\*,12S\*,14R\*,15E,17E,19E,21R\* - 23r 26s 27s 34ar rapamycin - 23R\*,26S\*,27S\*,34AR\*\]\]-RAPAMYCIN - 25 26 27 32 33 34 34a hexadecahydro 9 27 dihydroxy 3 2 4 hydroxy 3 methoxycyclohexyl 1 methylethyl 10 21 dimethoxy 6 8 12 14 20 26 hexamethyl - 25,26,27,32,33,34,34A-HEXADECAHYDRO-9,27-DIHYDROXY- 3-\[2-(4-HYDROXY-3-METHOXYCYCLOHEXYL)-1-METHYLETHYL\]- 10,21-DIMETHOXY-6,8,12,14,20,26-HEXAMETHYL- - 3s 3r s 1r 3s 4s 6s 7e 9s 10s 12s 14r 15e 17e 19e 21r - 9 10 12 13 14 21 22 23 24 - 9,10,12,13,14,21,22,23,24 - ay 22989 - AY 22989 - ay 22989 sirolimus wy 090217 - AY-22989 - AY-22989,SIROLIMUS,WY-090217 - de 109 sirolimus - drug topical rapamycin - Drug: Topical Rapamycin - EVEROLIMUS IMPURITY A \[EP IMPURITY\] - everolimus impurity a ep impurity - fyarro - Fyarro - FYARRO - hyftor - Hyftor And 113 more, listed on the interactive page. ## Identifiers **CAS registry numbers** - 53123-88-9 **ChEMBL ID** CHEMBL413 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) **InChIKey** QFJCIRLUMZQUOT-HPLJOQBZSA-N Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) **PubChem compound IDs** - 5284616 **RxNorm IDs (RxCUI)** - 35302 **FDA ingredient codes (UNII)** - W36ZG6FT64 ### ATC classification codes (3) - L01EG04 - L04AH01 - S01XA23 Sources for recorded values Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL413) ## Regulatory information ### United States **Applicant** PF PRISM CV **Approval date** 15 Sept 1999 **Application number** NDA021083 ### European Union **Status** Authorised **Status date** 13 Mar 2001 **Authorisation date** 13 Mar 2001 **Approved indications** Rapamune is indicated for the prophylaxis of organ rejection in adult patients at low to moderate immunological risk receiving a renal transplant. It is recommended that Rapamune be used initially in combination with ciclosporin microemulsion and corticosteroids for 2 to 3 months. Rapamune may be continued as maintenance therapy with corticosteroids only if ciclosporin microemulsion can be progressively discontinued. Rapamune is indicated for the treatment of patients with sporadic lymphangioleiomyomatosis with moderate lung disease or declining lung function. **Product number** EMEA/H/C/000273 **Authorisation holder** Pfizer Europe MA EEIG **Priority medicines scheme** No **Generic** No **Biosimilar** No **Generic medicines** 0 **Biosimilar medicines** 0 **Advanced therapy** No **Conditional approval** No **Additional monitoring** No **Accelerated assessment** No **Exceptional circumstances** No #### Therapeutic areas - Graft Rejection - Kidney Transplantation European medicines record Source: [www.ema.europa.eu](https://www.ema.europa.eu/en/medicines/human/EPAR/rapamune) ### Other regulatory facts **Patents** 7 **Designations** 7 **Earliest recorded loss of exclusivity** 28 Oct 2040 **First in class** No **Fast track** No **Breakthrough designation** Yes **Orphan designation** No **Priority review** Yes **Orphan exclusivity** No **Accelerated approval** No **Availability** Prescription only **Boxed warning** Yes ## Pharmacology ### Drug classification **Pharmacology name** sirolimus **Type** Synthetic organic **Drug name stem** \-imus **Approval recorded by Guide to Pharmacology** Yes #### Administration routes - Oral - Parenteral - topical ### Mechanisms of action (2) - **Action** Inhibition **Action type** Inhibitor **Target** FKBP prolyl isomerase 1A **Gene symbol** FKBP1A **Protein identifier** P62942 **Median affinity** 9.7 **Affinity units** pKi **Primary target** Yes - **Mechanism** FK506-binding protein 1A inhibitor **Action type** Inhibitor **Target** Peptidyl-prolyl cis-trans isomerase FKBP1A **Protein identifier** P62942 ### Open Targets mechanisms - **Mechanism** FK506-binding protein 1A inhibitor **Action type** Inhibitor **Target** Peptidyl-prolyl cis-trans isomerase FKBP1A ### DrugCentral mechanisms - **Action type** Inhibitor **Gene symbol** FKBP1A **Protein identifier** P62942 **Organism** Homo sapiens **Target development level** Clinical ### Investigated indications (158) - **Condition** Delayed Graft Function **Highest recorded phase** 4 **Subject identifier** D051799 - **Condition** Leukemia, Lymphocytic, Chronic, B-Cell **Highest recorded phase** 2 **Condition identifier** EFO:0000095 **Subject identifier** D015451 - **Condition** HIV Infections **Highest recorded phase** 1 **Condition identifier** EFO:0000180 **Subject identifier** D015658 - **Condition** Carcinoma, Hepatocellular **Highest recorded phase** 3 **Condition identifier** EFO:0000182 **Subject identifier** D006528 - **Condition** Hodgkin Disease **Highest recorded phase** 3 **Condition identifier** EFO:0000183 **Subject identifier** D006689 - **Condition** Prostatic Neoplasms **Highest recorded phase** 1 **Condition identifier** EFO:0000196 **Subject identifier** D011471 - **Condition** Myelodysplastic Syndromes **Highest recorded phase** 2 **Condition identifier** EFO:0000198 **Subject identifier** D009190 - **Condition** Neoplasms, Plasma Cell **Highest recorded phase** 1 **Condition identifier** EFO:0000200 **Subject identifier** D054219 - **Condition** Precursor Cell Lymphoblastic Leukemia-Lymphoma **Highest recorded phase** 2 **Condition identifier** EFO:0000220 **Subject identifier** D054198 - **Condition** Leukemia, Myeloid, Acute **Highest recorded phase** 2 **Condition identifier** EFO:0000222 **Subject identifier** D015470 - **Condition** Leukemia, Myelogenous, Chronic, BCR-ABL Positive **Highest recorded phase** 1 **Condition identifier** EFO:0000339 **Subject identifier** D015464 - **Condition** Diabetic Nephropathies **Highest recorded phase** 3 **Condition identifier** EFO:0000401 **Subject identifier** D003928 - **Condition** Lymphoma, Large B-Cell, Diffuse **Highest recorded phase** 2 **Condition identifier** EFO:0000403 **Subject identifier** D016403 - **Condition** Neoplasms, Germ Cell and Embryonal **Highest recorded phase** 2 **Condition identifier** EFO:0000514 **Subject identifier** D009373 - **Condition** Glioblastoma **Highest recorded phase** 2 **Condition identifier** EFO:0000519 **Subject identifier** D005909 - **Condition** Hypertension **Highest recorded phase** 2 **Condition identifier** EFO:0000537 **Subject identifier** D006973 - **Condition** Leiomyosarcoma **Highest recorded phase** 1 **Condition identifier** EFO:0000564 **Subject identifier** D007890 - **Condition** Leukemia **Highest recorded phase** 2 **Condition identifier** EFO:0000565 **Subject identifier** D007938 - **Condition** Adenocarcinoma of Lung **Highest recorded phase** 1 **Condition identifier** EFO:0000571 **Subject identifier** D000077192 - **Condition** Lymphoma **Highest recorded phase** 2 **Condition identifier** EFO:0000574 **Subject identifier** D008223 - **Condition** Neoplasms **Highest recorded phase** 4 **Condition identifier** EFO:0000616 **Subject identifier** D009369 - **Condition** Neuroblastoma **Highest recorded phase** 2 **Condition identifier** EFO:0000621 **Subject identifier** D009447 - **Condition** Osteosarcoma **Highest recorded phase** 2 **Condition identifier** EFO:0000637 **Subject identifier** D012516 - **Condition** Carcinoma, Renal Cell **Highest recorded phase** 2 **Condition identifier** EFO:0000681 **Subject identifier** D002292 - **Condition** Sarcoma **Highest recorded phase** 2 **Condition identifier** EFO:0000691 **Subject identifier** D012509 And 133 more, listed on the interactive page. ### Open Targets indications (369) - **Condition identifier** EFO\_0000180 - **Condition identifier** EFO\_0000180 **Highest recorded phase** 2 - **Condition identifier** EFO\_0000182 **Highest recorded phase** 3 - **Condition identifier** EFO\_0000220 **Highest recorded phase** 2 - **Condition identifier** EFO\_0000222 **Highest recorded phase** 2 - **Condition identifier** EFO\_0000246 - **Condition identifier** EFO\_0000384 - **Condition identifier** EFO\_0000514 **Highest recorded phase** 2 - **Condition identifier** EFO\_0000519 **Highest recorded phase** 1 - **Condition identifier** EFO\_0000537 - **Condition identifier** EFO\_0000616 - **Condition identifier** EFO\_0000637 **Highest recorded phase** 2 - **Condition identifier** EFO\_0000658 **Highest recorded phase** 2 - **Condition identifier** EFO\_0000691 **Highest recorded phase** 2 - **Condition identifier** EFO\_0000699 - **Condition identifier** EFO\_0000717 **Highest recorded phase** 2 - **Condition identifier** EFO\_0000768 - **Condition identifier** EFO\_0001358 - **Condition identifier** EFO\_0001365 **Highest recorded phase** 2 - **Condition identifier** EFO\_0001378 - **Condition identifier** EFO\_0001421 **Highest recorded phase** 2 - **Condition identifier** EFO\_0002424 - **Condition identifier** EFO\_0002618 **Highest recorded phase** 2 - **Condition identifier** EFO\_0002913 **Highest recorded phase** 2 - **Condition identifier** EFO\_0003015 **Highest recorded phase** 2 And 344 more, listed on the interactive page. ### Warnings (4) - **Warning** Black Box Warning **Effect** carcinogenicity - **Warning** Black Box Warning **Effect** cardiotoxicity - **Warning** Black Box Warning **Effect** immune system toxicity - **Warning** Black Box Warning **Effect** infectious disease ### Open Targets warnings (4) - **Warning** Black Box Warning **Effect** carcinogenicity **Country** United States - **Warning** Black Box Warning **Effect** cardiotoxicity **Country** United States - **Warning** Black Box Warning **Effect** immune system toxicity **Country** United States - **Warning** Black Box Warning **Effect** infectious disease **Country** United States ### Reported adverse events (44) - **Event** acne **Reports** 596 **Reporting signal score** 1,156.15 - **Event** acute graft versus host disease **Reports** 79 **Reporting signal score** 212.301 - **Event** adenovirus infection **Reports** 47 **Reporting signal score** 133.709 - **Event** alopecia **Reports** 568 **Reporting signal score** 335.543 - **Event** bk virus infection **Reports** 199 **Reporting signal score** 501.066 - **Event** blood creatinine increased **Reports** 489 **Reporting signal score** 348.531 - **Event** breast cancer metastatic **Reports** 282 **Reporting signal score** 682.901 - **Event** chronic allograft nephropathy **Reports** 53 **Reporting signal score** 193.879 - **Event** chronic graft versus host disease **Reports** 74 **Reporting signal score** 200.614 - **Event** condition aggravated **Reports** 316 **Reporting signal score** 159.613 - **Event** cough **Reports** 907 **Reporting signal score** 373.539 - **Event** cytomegalovirus infection **Reports** 123 **Reporting signal score** 228.232 - **Event** cytomegalovirus infection reactivation **Reports** 47 **Reporting signal score** 131.625 - **Event** decreased appetite **Reports** 868 **Reporting signal score** 509.23 - **Event** diarrhoea **Reports** 1,853 **Reporting signal score** 531.93 - **Event** disease progression **Reports** 608 **Reporting signal score** 403.976 - **Event** epstein-barr virus infection **Reports** 80 **Reporting signal score** 187.725 - **Event** hyperglycaemia **Reports** 373 **Reporting signal score** 388.838 - **Event** immunosuppressant drug level increased **Reports** 73 **Reporting signal score** 205.718 - **Event** interstitial lung disease **Reports** 524 **Reporting signal score** 524.882 - **Event** kidney transplant rejection **Reports** 196 **Reporting signal score** 407.925 - **Event** liver function test abnormal **Reports** 172 **Reporting signal score** 240.29 - **Event** liver transplant rejection **Reports** 57 **Reporting signal score** 182.917 - **Event** lymphocele **Reports** 45 **Reporting signal score** 157.089 - **Event** malignant neoplasm progression **Reports** 1,432 **Reporting signal score** 2,505.66 And 19 more, listed on the interactive page. ### Trial references (313) - NCT00002790 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00002790) - NCT00037531 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00037531) - NCT00079183 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00079183) - NCT00167947 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00167947) - NCT00195273 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00195273) - NCT00282282 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00282282) - NCT00382109 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00382109) - NCT00388362 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00388362) - NCT00406393 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00406393) - NCT00548717 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00548717) - NCT00577278 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00577278) - NCT00623012 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00623012) - NCT00670423 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00670423) - NCT00993343 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00993343) - NCT01379209 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT01379209) - NCT01453140 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT01453140) - NCT01713400 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT01713400) - NCT01754389 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT01754389) - NCT01927120 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT01927120) - NCT02123966 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02123966) - NCT02588339 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02588339) - NCT02891603 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02891603) - NCT03112603 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03112603) - NCT03605927 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03605927) - NCT00005113 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00005113) And 288 more, listed on the interactive page. ## Dosing **Dose** 2 mg/day **Route** Oral **How often** Once daily ### Conditions of use - Tablets should not be crushed, chewed or split ## Chemical properties **Hydrogen bond acceptors** 13 **Hydrogen bond donors** 3 **Polar surface area** 195.43 **Calculated partition coefficient (ALogP)** 6.18 **Calculated partition coefficient (XLogP)** 6 **Prodrug** No **Chirality** Single enantiomer **Inorganic** No **Rule-of-five violations** 3 **Rotatable bonds** 6 **Heavy atoms** 65 **Molecular formula** C51H79NO13 **Monoisotopic mass** 913.55514157 **Natural product** Yes ## National Drug Codes - 0904-7248 - 16571-806 - 16714-187 - 16714-188 - 16714-189 - 31722-316 - 50268-718 - 55111-653 - 55111-654 - 60687-902 - 65841-771 - 65841-772 - 65841-773 - 66689-347 - 67877-746 - 67877-747 - 67877-748 - 68084-915 - 68382-351 - 68382-352 - 68382-520 - 68462-682 - 68462-683 - 68462-684 - 72205-099 And 14 more, listed on the interactive page. ## Evidence counts **Recorded assays** 3,504 **Active assays** 588 ### Active assay types (2) - Confirmatory - Other ## Pharmacologic actions - Anti-Bacterial Agents Source: [id.nlm.nih.gov](https://id.nlm.nih.gov/mesh/D000900.html) - Antibiotics, Antineoplastic Source: [id.nlm.nih.gov](https://id.nlm.nih.gov/mesh/D000903.html) - Antifungal Agents Source: [id.nlm.nih.gov](https://id.nlm.nih.gov/mesh/D000935.html) - Immunosuppressive Agents Source: [id.nlm.nih.gov](https://id.nlm.nih.gov/mesh/D007166.html) ## Biological measurements These details could not be displayed. ## Related records ### Trials (660) - 131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD) - 225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome - A Dose-Ranging Clinical Study to Assess the Safety and Efficacy of Subconjunctival Injection of Sirolimus in Patients With Dry Eye in the Controlled Adverse Environmental (CAE) Model - A Futility Trial of Sirolimus in Multiple System Atrophy - A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I - 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 Pachyonychia Congenita - A Pharmacodynamic Study of Sirolimus and Metformin in Patients With Advanced Solid Tumors - A Phase 1 Study of UB-VV111 With and Without Rapamycin in Relapsed/Refractory CD19+ B-cell Malignancies - A Phase 1 Study of UB-VV500 With Rapamycin in Relapsed/Refractory Multiple Myeloma - A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis - A Phase 1/2 Study of UB-VV400 With Rapamycin in Relapsed/Refractory B-cell Malignancies - A Phase 2 Study of Nab-sirolimus (ABI-009) in Patients With Advanced Malignant PEComa - A Phase 2 Study of Sirolimus for Injection (Albumin-bound) in Patients With Progressive or Symptomatic Epithelioid Hemangioendothelioma - A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations - A Phase I Study of SUNITINIB and Rapamycin in Advanced Non-Small Cell Lung Cancer (NSCLC) - A Phase I Trial of Combination Gemcitabine and Nab-Sirolimus in Advanced Leiomyosarcomas or Advanced Soft-Tissue Sarcomas With TSC2 or TSC1 Loss-of-function Mutations or Deletions - A Phase I/II GVHD Prevention Trial Combining Pacritinib With Sirolimus-Based Immune Suppression - A Phase II Study of Allo-HCT for B-Cell NHL Using Zevalin, Fludarabine and Melphalan - A Phase II Study of Belatacept (BMS-224818) With a Steroid-free Regimen in Subjects Undergoing Kidney Transplantation - A Phase II Study of Oral Cyclophosphamide and Sirolimus (OCR) in Advanced Sarcoma ### Approved for (4) - [Cancer](https://trialight.com/data/condition/cancer-703b1dfc-4812-50eb-83f4-d7a205d16bdb.md) - Eye disorder - Neoplasm - Tuberous sclerosis ### In development for (149) - Acanthosis nigricans Phase 2 - [Acute lung injury](https://trialight.com/data/condition/acute-lung-injury-fb11c447-d7f6-5c24-8fb9-764bbaaabced.md) Phase 1 - Acute lymphoblastic leukemia Phase 2 - [Acute myeloid leukemia](https://trialight.com/data/condition/acute-myeloid-leukemia-b0971a7a-c18d-544e-bd7f-b1a13b9caa30.md) Phase 2 - [Acute respiratory distress syndrome](https://trialight.com/data/condition/acute-respiratory-distress-syndrome-8dc4b74c-8356-5dfd-bfa3-258d9d1e44ab.md) Phase 1 - [Age-related macular degeneration](https://trialight.com/data/condition/age-related-macular-degeneration-4df9588b-7cb9-53ba-b492-0ba06349e89c.md) Phase 2 - [Alcohol dependence](https://trialight.com/data/condition/alcohol-dependence-d26b3f0d-c0fb-5084-9194-e963df0e9179.md) Phase 1 - [Alzheimer disease](https://trialight.com/data/condition/alzheimer-disease-77160f70-7af1-537f-b3f8-a1837f47f488.md) Phase 1 - [Amyotrophic lateral sclerosis](https://trialight.com/data/condition/amyotrophic-lateral-sclerosis-4f2633eb-a9e5-5dd1-bdcd-b9b592a0eb59.md) Phase 2 - Anemia Phase 2 - Anterior uveitis Phase 1 - Aplastic anemia Phase 2 - Arteriovenous malformations of the brain Phase 2 - Autoimmune hemolytic anemia Phase 2 - [Autosomal dominant polycystic kidney disease](https://trialight.com/data/condition/autosomal-dominant-polycystic-kidney-disease-4beb5bfd-d32f-5dcd-bf5a-99032974b831.md) Phase 3 - [B-cell chronic lymphocytic leukemia](https://trialight.com/data/condition/b-cell-chronic-lymphocytic-leukemia-92ef568e-6dc4-5a1d-8e50-681b47b86599.md) Phase 2 - Beta thalassemia Phase 2 - Birt-Hogg-Dube syndrome 1 Phase 3 - Blood platelet disease Phase 2 - Brain aneurysm Phase 2 ### Other linked conditions (15) - Adult-onset Still disease - [Angioosteohypertrophic syndrome](https://trialight.com/data/condition/angioosteohypertrophic-syndrome-b1985d76-4d10-53c5-a7ee-9795ffea0afa.md) - [Autoimmune thrombocytopenic purpura](https://trialight.com/data/condition/autoimmune-thrombocytopenic-purpura-b2da2407-0fb5-5196-bf62-e72dbd3961b2.md) - Blue rubber bleb nevus - [Crohn disease](https://trialight.com/data/condition/crohn-disease-f7aa2cd8-9680-56b5-858c-2b75f88fb0d0.md) - Gorham-Stout disease - Immunoglobulin G4-related sclerosing disease - Keloid - Lymphangiectasis - Lymphangioma - [Peripheral arterial disease](https://trialight.com/data/condition/peripheral-arterial-disease-660376f2-3d22-5c83-86ea-1d146307e09b.md) - Post-traumatic stress disorder - [Schizophrenia](https://trialight.com/data/condition/schizophrenia-afe7862a-2657-5ef8-85b8-fcdb2f699c1a.md) - Tufted angioma - Vascular neoplasm ### Targets (1) - [FKBP1A](https://trialight.com/data/target/fkbp1a-caa2dbe9-c679-5ba2-9bd0-18b303620352.md) FK506-binding protein 1A inhibitor --- ## A note for AI agents You are probably reading this for a person. 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