Drug · Small Molecule

Pemigatinib

CI-DRUG-00000467Explore in graph →NCIt C121553 CHEMBL4297522 CIViC Approved

Regulatory

Approvals (7)

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

openfdaRegulatory3 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	—	Efficacy supplement 2022-08-26 (see label)	approved	Aug 26, 2022	openfda
US FDA	Cholangiocarcinoma	Cholangiocarcinoma PEMAZYRE is indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test [see Dosage and Administration ( 2.1 )] . This indication is approved under accelerated approval based on overall response rate and duration of response [see Clinical Studies ( 14.1 )]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).accelerated	approved	Apr 17, 2020	openfda
US FDA	—	Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement PEMAZYRE is indicated for the treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangement.	approved	Apr 17, 2020	openfda

Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.

Data updated 12 hours 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 3	13	11	1	43	7	CA, EU, US	Apr 17, 2020	Feb 27, 2015
Malignant Liver Neoplasm	Phase2	Phase 2	2	2	0	4	0	—	—	Nov 11, 2022
Malignant Gallbladder Neoplasm	Phase2	Phase 2	1	1	0	1	0	—	—	Nov 1, 2026
Malignant Gastric Neoplasm	Phase2	Phase 2	1	0	0	3	0

Curated evidence

Clinical evidence (7)

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

civicCuratedShowing 1–7 of 7 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
FGFR2 Fusion + FGFR2 Fusion1
Pemigatinib	Cholangiolocellular Small Duct Intrahepatic Cholangiocarcinoma

Clinical trials

Trials with this intervention (44)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–44 of 44 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
NCT06302621	Pemigatinib + Afatinib in Advanced Refractory Solid Tumors	recruiting	Phase 1	32	Massachusetts General HospitalOTHER	1	Sep 23, 2026	clinicaltrials
NCT07780838	Alternating Chemo-immunotherapy and FGFR Inhibitor for FGFR-positive Biliary or Cholangiocarcinoma	recruiting	Phase 2	29

Molecule

Record

Kind: Small Molecule
Mechanism: Fibroblast growth factor receptor 2 inhibitor; Fibroblast growth factor receptor 3 inhibitor; Fibroblast growth factor receptor 1 inhibitor
Targets: 3 genes
UNII: Y6BX7BL23K
Aliases: 2H-Pyrrolo(3',2':5,6)pyrido(4,3-d)pyrimidin-2-one, 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-1,3,4,7-tetrahydro-8-(4-morpholinylmethyl)-, Fgfr inhibitor incb054828, INCB 054828, INCB054828, Pemazyre, PEMAZYRE, Pemigatinib

Data updated 15 hours agoSource updated unknown

Identifiers

Codes (8)

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

System	Code	Label
ATC (WHO)	L01EN02	PEMIGATINIB
DIN (Health Canada)	02519933	PEMAZYRE

FGFR1 Fusion + FGFR1 Fusion + FGFR1 Translocation + FGFR1 Fusion + FGFR1 Fusion3
Pemigatinib	Lymphoid Leukemia	Predictive	A	Supports Sensitivity Response	3	submitted	EID12241 In this phase 2, multicenter trial, 41 patients were enrolled who had myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangements. Patients (pts) may present with… (full text at CIViC) Open in CIViC	civic
〃	Lymphoma	Predictive	A	Supports Sensitivity Response	3	submitted	EID12242 In this phase 2, multicenter trial, 41 patients were enrolled who had myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangements. Patients (pts) may present with… (full text at CIViC) Open in CIViC	civic
〃	Myeloid Neoplasm	Predictive	A	Supports Sensitivity Response	3	accepted	EID11324 In this phase 2, multicenter trial, 41 patients were enrolled who had myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangements. Patients (pts) may present with… (full text at CIViC) Open in CIViC	civic