Drug · Small Molecule

Sirolimus

CI-DRUG-00000554Explore in graph →NCIt C1212 CHEMBL413 CIViC Approved

Regulatory

Approvals (16)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

openfdaRegulatory16 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	—	HYFTOR is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis in adults and pediatric patients 6 years of age and older. HYFTOR is an mTOR inhibitor immunosuppressant indicated for the treatment of facial angiofibroma associated with tuberous sclerosis in adults and pediatric patients 6 years of age and older. ( 1 )	approved	Mar 22, 2022	openfda
US FDA

Data updated 17 days agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	52	26	24	228	16	US	Sep 15, 1999	1996-03
Skin Carcinoma	Phase4	Phase 4	3	2	0	5	0	—	—	2013-04
Malignant Uterine Corpus Neoplasm	Phase4	Phase 4	0	0	0	2	0	—	—	2012-03
Leukemia	Phase3	Phase 3	13	6	4	65	0	—	—

Curated evidence

Clinical evidence (32)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–32 of 32 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
CRLF2 Rearrangement1
Sirolimus	Acute Lymphoblastic Leukemia

Clinical trials

Trials with this intervention (230)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 201–230 of 230 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT00707135	Rapamycin in Advanced Cancers	completed	Phase 1	40	University of ChicagoOTHER	1	Jan 17, 2014	clinicaltrials
NCT00708591	Study of Rapamycin Plus Ketoconazole in Advanced Cancers	completed	Phase 1	57	University of Chicago

Molecule

Record

Kind: Small Molecule
Mechanism: FK506-binding protein 1A inhibitor
Targets: 1 gene
UNII: W36ZG6FT64
Aliases: 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-, [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, 9,10,12,13,14,21,22,23,24,25,26,27,32,33,34,34a-Hexadecahydro-9,27-dihydroxy-3-((1R)-2-((1S,3R,4R)-4-hydroxy-3-methoxycyclohexyl)-1-methylethyl)-10,21-dimethoxy-6,8,12,14,20,26-hexamethyl-23,27-epoxy-3H-pyrido(2,1-c)(1,4)oxaazacyclohentriacontine-1,5,11,2, AY 22989, AY-22989, AY22989, AY-22989,SIROLIMUS,WY-090217, Fyarro, Hyftor, L04AA10, Npc-12g, NSC-226080, NSC-2260804, NSC-2260804,AY-22989, RAPA, Rapalimus, Rapamune, RAPAMYCIN, RAPAMYCIN (AY-22989), RAPAMYCIN (SIROLIMUS), SILA 9268A, SILA9268A, Sirolimus, Sirolimus component of nab-rapamycin rapamycin, SIROLIMUS COMPONENT OF NAB-RAPAMYCIN RAPAMYCIN, SIROLIMUS COMPONENT OF SM-88, SIROLIMUSRAPAMUNERAPAMYCIN(-)-RAPAMYCINANTIBIOTIC AY 22989SIIA 9268AAY 22989WY 09021723,27-EPOXY-3H-PYRIDO[2,1-C][1,4]OXAAZACYCLOHENTRIACONTINE- 1,5,11,28,29(4H,6H,31H)-PENTONE, WY 090217 … (+2)

Data updated 18 hours agoSource updated unknown

Identifiers

Codes (0)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

ERBB2 Y772_A775DUP2
Afatinib + SirolimusCombination	Lung Carcinoma	Predictive	D	Supports Sensitivity Response	4	rejected	EID1046 In mice, human HER2 with YVMA insertion was placed under a doxycycline-inducible, clara cell specific promoter. Induction of mutant HER2 in these mice drove rapid development of adenosquamous lung tum… (full text at CIViC) PMID 19122144 · Perera et al., 2009 · Open in CIViC	civic
〃	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	accepted	EID960 In preclinical studies of transgenic mice and xenografts, continuous expression of mutant ERBB2 (M774insAYVM or the equivalent A775insYVMA) was found to be essential for tumor maintenance. Furthermore… (full text at CIViC) PMID 19122144 · Perera et al., 2009 · Open in CIViC	civic

STK11 Loss3
Everolimus + SirolimusSubstitutes	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	D	Does Not Support Sensitivity Response	3	accepted	EID1620 Preclinical study in 5 NSCLC cell lines. Two LKB1 (STK11) mutant cell lines had higher basal levels of mTOR activity (pS6K and p4EBP1 levels) but were not more sensitive to mTOR inhibitors (everolimus… (full text at CIViC) PMID 26027660 · Xiao et al., 2015 · Open in CIViC	civic
Sirolimus	Peutz-Jeghers SyndromeUNRESOLVED	Predictive	D	Supports Sensitivity Response	1	accepted	EID1618 Preclinical study in a mouse model of Peutz-Jeghers syndrome. Heterozygous Lkb1 (STK11) knockout mice were generated and treated with rapamycin after the onset of polyposis at 9 months of age. Rapamyc… (full text at CIViC) PMID 18281551 · Wei et al., 2008 · Open in CIViC	civic
〃	Peutz-Jeghers SyndromeUNRESOLVED	Predictive	D	Supports Sensitivity Response	1	accepted	EID1619 Treatment of mice with a heterozygous loss of Lkb1 (STK11) with rapamycin led to reduction in polyp size (p < 0.0001) and number of polyps (p=0.00022). pS6 staining was also reduced after rapamycin tr… (full text at CIViC) PMID 19541609 · Shackelford et al., 2009 · Open in CIViC	civic

STK11 Loss5
Sirolimus	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	—	submitted	EID2314 STK11 is mutated and inactivated in 20-30% of non-small cell lung cancers, most often in the adenocarcinoma subtype. PMID 15021901 · Carretero et al., 2004 · Open in CIViC	civic
〃	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	—	rejected	EID2315 STK11 is mutated and inactivated in 20-30% of non-small cell lung cancers, most often in the adenocarcinoma subtype. PMID 21102257 · Marcus et al., 2010 · Open in CIViC	civic
〃	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	—	submitted	EID2316 STK11 is mutated and inactivated in 20-30% of non-small cell lung cancers, most often in the adenocarcinoma subtype. PMID 12097271 · Sanchez-Cespedes et al., 2002 · Open in CIViC	civic
〃	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	—	submitted	EID2317 STK11 is mutated and inactivated in 20-30% of non-small cell lung cancers, most often in the adenocarcinoma subtype. PMID 18948947 · Ding et al., 2008 · Open in CIViC	civic
〃	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	—	submitted	EID2313 NSCLC cell lines that contain inactivating STK11 mutations and activating KRAS mutations have shown a reduced proliferation rate in response to the MEK inhibitor CI-1040 (P<0.001) compared to NSCLC ce… (full text at CIViC) PMID 19165201 · Mahoney et al., 2009 · Open in CIViC	civic

MTOR M2327I3
RapaLink-1 + SirolimusSubstitutes	Malignant Breast Neoplasm	Predictive	D	Does Not Support Resistance	3	accepted	EID1545 MCF-7 breast cancer cell line was exposed to mTOR inhibitor AZD8055 until resistance developed. Deep sequencing revealed an MTOR M2327I mutation in the AZD8055-resistant clone. These cells remained se… (full text at CIViC) PMID 27279227 · Rodrik-Outmezguine et al., 2016 · Open in CIViC	civic
Sirolimus	Malignant Breast Neoplasm	Predictive	D	Supports Resistance	4	rejected	EID1546 MCF-7 breast cancer cell line was exposed to mTOR inhibitor rapamycin until resistance developed. Deep sequencing revealed an MTOR A2034V mutation in the resistant clone. Downstream signalling was ind… (full text at CIViC) PMID 27279227 · Rodrik-Outmezguine et al., 2016 · Open in CIViC	civic
RapaLink-1 + SirolimusSubstitutes	Malignant Breast Neoplasm	Predictive	D	Supports Sensitivity Response	4	rejected	EID10082 MCF-7 breast cancer cell line was exposed to mTOR inhibitor AZD8055 until resistance developed. Deep sequencing revealed an MTOR M2327I mutation in the resistant clone (TKi-R). A novel mTOR inhibitor … (full text at CIViC) PMID 27279227 · Rodrik-Outmezguine et al., 2016 · Open in CIViC	civic