Palifermin

Summary

Palifermin (CHEMBL1201821) is an approved protein (ATC V03AF08) targeting FGFR2; indicated across 30 conditions including mucositis and stomatitis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Protein
• ATC class: V03AF08
• Targets: 1 (FGFR2)
• Indications: 30 conditions
• Clinical trials: 52

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201821
Name	Palifermin
Type	Protein
Max phase	4
ATC	V03AF08

Also known as: Kepivance, Palifermin, Palifermina, Palifermine, PALIFERMIN

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
FGFR2	fibroblast growth factor receptor 2	Agonist		1.7%	P21802

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): FGFR2.

Top Reactome pathways

18 total, by targets touching each:

Pathway	Targets	Genes
PI3K Cascade	1	FGFR2
PIP3 activates AKT signaling	1	FGFR2
FGFR2c ligand binding and activation	1	FGFR2
FGFR2b ligand binding and activation	1	FGFR2
Signaling by FGFR2 amplification mutants	1	FGFR2
Activated point mutants of FGFR2	1	FGFR2
Constitutive Signaling by Aberrant PI3K in Cancer	1	FGFR2
Phospholipase C-mediated cascade; FGFR2	1	FGFR2
PI-3K cascade:FGFR2	1	FGFR2
SHC-mediated cascade:FGFR2	1	FGFR2
FRS-mediated FGFR2 signaling	1	FGFR2
Negative regulation of FGFR2 signaling	1	FGFR2
Signaling by FGFR2 in disease	1	FGFR2
RAF/MAP kinase cascade	1	FGFR2
FGFR2 alternative splicing	1	FGFR2
PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling	1	FGFR2
Signaling by FGFR2 IIIa TM	1	FGFR2
Signaling by FGFR2 fusions	1	FGFR2

Dominant GO biological processes

GO term	Targets
negative regulation of transcription by RNA polymerase II	1
angiogenesis	1
ureteric bud development	1
in utero embryonic development	1
epithelial to mesenchymal transition	1
positive regulation of mesenchymal cell proliferation	1
outflow tract septum morphogenesis	1
membranous septum morphogenesis	1
endochondral bone growth	1
apoptotic process	1
cell-cell signaling	1
axonogenesis	1
positive regulation of cell population proliferation	1
fibroblast growth factor receptor signaling pathway	1
regulation of smoothened signaling pathway	1

Indications & clinical

Indications

30 indications (3 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
mucositis	4	MONDO:0020579	EFO:1001898
stomatitis	4	MONDO:0004842	EFO:1001904
leukemia	3	MONDO:0005059	EFO:0000565
lymphoma	3	MONDO:0005062	EFO:0000574
plasma cell myeloma	3	MONDO:0009693	EFO:0001378
head and neck cancer	3	MONDO:0005627	EFO:0006859
squamous cell carcinoma	3	MONDO:0005096	EFO:0000707
neoplasm	3	MONDO:0005070	MONDO:0004992
HIV infectious disease	2	MONDO:0005109	EFO:0000764
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
T-cell non-Hodgkin lymphoma	2	MONDO:0015760	MONDO:0015760
colonic neoplasm	2	MONDO:0005401	MONDO:0021063
asthma	2	MONDO:0004979	MONDO:0004979
Hodgkins lymphoma	1	MONDO:0004952	EFO:0000183
Stevens-Johnson syndrome	1	MONDO:0018229	EFO:0004276
non-Hodgkin lymphoma	1	MONDO:0018908	EFO:0005952
plasma cell leukemia	1	MONDO:0018689	EFO:0006475
combined immunodeficiency, X-linked	1	MONDO:0010730	EFO:1001451
T-B+ severe combined immunodeficiency due to gamma chain deficiency	1	MONDO:0010315	EFO:0005555
graft versus host disease	1	MONDO:0013730	MONDO:0013730
hematopoietic and lymphoid system neoplasm	1	MONDO:0002334	MONDO:0044881
multiple sclerosis	1	MONDO:0005301	MONDO:0005301
toxic epidermal necrolysis	1	MONDO:0019810	EFO:0004775
chronic granulomatous disease	1	MONDO:0018305	MONDO:0018305
neuroepithelial neoplasm	0	MONDO:0021193	MONDO:0021193
epidermolysis bullosa	0	MONDO:0006541	EFO:1000690
alopecia	0	MONDO:0004907	MONDO:0004907

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 52.

Phase distribution

Phase	Trials
PHASE2	17
PHASE1/PHASE2	13
PHASE1	11
PHASE3	5
Not specified	3
PHASE4	1
PHASE2/PHASE3	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00352703	PHASE4	COMPLETED	PROMPT - Palifermin in Reduction of Oral Mucositis in PBSC Transplantation
NCT00101582	PHASE3	COMPLETED	Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer
NCT00109031	PHASE3	COMPLETED	Palifermin for the Reduction of Oral Mucositis in Single-dose Evaluation (PROMISE)
NCT00131638	PHASE3	COMPLETED	A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Advanced Head and Neck Cancer
NCT00360971	PHASE3	TERMINATED	Palifermin in Lessening Oral Mucositis in Patients Undergoing Radiation Therapy and Chemotherapy for Locally Advanced Head and Neck Cancer
NCT00393822	PHASE2/PHASE3	COMPLETED	A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Stage 2B or 3 Locally Advanced, Colon Cancer
NCT02313792	PHASE3	UNKNOWN	Palifermin for Patients Receiving Hematopoietic Stem Cell Transplantation
NCT01306019	PHASE1/PHASE2	RECRUITING	Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
NCT01746849	PHASE2	ACTIVE_NOT_RECRUITING	Palifermin With Leuprolide Acetate for the Promotion of Immune Recovery Following Total Body Irradiation Based T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation
NCT06325709	PHASE1/PHASE2	RECRUITING	Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
NCT06851767	PHASE1/PHASE2	ENROLLING_BY_INVITATION	Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
NCT06959771	PHASE1/PHASE2	RECRUITING	Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
NCT06986382	PHASE1/PHASE2	NOT_YET_RECRUITING	Phase 1b/2a Trial of Allogeneic HSCT From an HLA-partially Matched Related or Unrelated Donor After TCRab+ T-cell/CD19+ B-cell Depletion for Patients With Monogenic and/or Early-onset Medically Refractory Crohn Disease
NCT07582172	PHASE2	NOT_YET_RECRUITING	Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome
NCT00004061	PHASE2	COMPLETED	Biological Therapy in Treating Patients Undergoing Radiation Therapy, Chemotherapy, and Peripheral Stem Cell Transplantation for Hematologic Cancer
NCT00004132	PHASE2	COMPLETED	Growth Factor to Prevent Oral Mucositis in Patients With Hematologic Cancer
NCT00094861	PHASE2	COMPLETED	Study to Evaluate Palifermin in the Reduction of Dysphagia in Patients With Locally Advanced Non-Small Cell Lung Cancer (NSCLC)
NCT00189488	PHASE2	COMPLETED	Trial to Evaluate Palifermin in the Reduction of Acute Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Allogeneic Marrow/Peripheral Blood Progenitor Cell (PBPC) Transplantation
NCT00267046	PHASE2	COMPLETED	Evaluate PKs and Efficacy Assessment of Palifermin in Patients With Sarcoma
NCT00376935	PHASE2	COMPLETED	Effectiveness of Palifermin in Increasing CD4 Counts in Treatment-Experienced HIV Infected Adults
NCT00540332	PHASE1/PHASE2	WITHDRAWN	A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Locally Advanced Head and Neck Cancer Receiving Postoperative Radiotherapy
NCT00570999	PHASE2	WITHDRAWN	Palifermin After Haploidentical PBSCT
NCT00593554	PHASE2	TERMINATED	Phase 2 Haplotype Mismatched HSCT in Patients With Hematological Malignancies
NCT00626639	PHASE1/PHASE2	COMPLETED	A Study of Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer Receiving Postoperative Radiotherapy and Concurrent Chemotherapy
NCT00629798	PHASE2	COMPLETED	Busulfan, Melphalan, and Fludarabine With Peri-transplant Palifermin, Followed by a T-Cell Depleted Hematopoietic Stem Cell Transplant From HLA Matched or Mismatched Related or Unrelated Donors in Patients With Advanced Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)
NCT00728585	PHASE2	WITHDRAWN	Palifermin in Preventing Oral Mucositis Caused by Chemotherapy and/or Radiation Therapy in Young Patients Undergoing Stem Cell Transplant
NCT01200329	PHASE2	COMPLETED	High-Dose Gemcitabine, Busulfan and Melphalan for Patients With Refactory Hodgkin’s Disease
NCT01237951	PHASE2	COMPLETED	High-Dose Gemcitabine, Busulfan and Melphalan With Hematopoietic-Cell Support for Patients With Poor-Risk Myeloma
NCT01712945	PHASE1/PHASE2	TERMINATED	Keratinocyte Growth Factor to Prevent Autoimmunity After Alemtuzumab Treatment of Multiple Sclerosis
NCT01746173	PHASE2	TERMINATED	CHOEP + High Dose Therapy + Auto SCT for T-Cell Lymphoma
NCT02037347	PHASE1/PHASE2	TERMINATED	Study to Evaluate the Use of Palifermin to Treat Toxic Epidermal Necrolysis
NCT02114502	PHASE2	WITHDRAWN	Carfilzomib/SAHA Combined With High-Dose Gemcitabine/Busulfan/Melphalan With Autologous Stem Cell Transplant in Myeloma
NCT02356159	PHASE1/PHASE2	COMPLETED	Study of Palifermin (Kepivance) in Persons Undergoing Unrelated Donor Allogeneic Hematopoietic Cell Transplantation
NCT02589145	PHASE1/PHASE2	TERMINATED	Lenalidomide Combined With Vorinostat/Gemcitabine/Busulfan/Melphalan With Autologous Stem-Cell Transplantation in Diffuse Large B-Cell Lymphoma of the ABC Subtype
NCT02961816	PHASE2	WITHDRAWN	Panobinostat Combined With High-Dose Gemcitabine/Busulfan/Melphalan With Autologous Stem Cell Transplant for Patients With Refractory/Relapsed Lymphoma
NCT03100877	PHASE1/PHASE2	WITHDRAWN	Melphalan, Total Marrow Irradiation, and Autologous Stem Cell Transplantation in Treating Patients With High-Risk Multiple Myeloma
NCT03315078	PHASE1/PHASE2	UNKNOWN	Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency
NCT05139004	PHASE1	ACTIVE_NOT_RECRUITING	90Y-DOTA-anti-CD25 Basiliximab, Fludarabine, Melphalan, and Total Marrow and Lymphoid Irradiation for the Treatment of High-Risk Acute Leukemia or Myelodysplastic Syndrome
NCT00070616	PHASE1	COMPLETED	Pharmacokinetic Study of Palifermin in Subjects Receiving Radiation Therapy and Chemotherapy Followed by Blood Stem Cell Support
NCT00361348	PHASE1	COMPLETED	Palifermin DDI (Drug Drug Interaction)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

53 molecules share ≥1 primary target. Top 53 by shared-target count:

Molecule	Source	Status	Shared targets
PAZOPANIB	ChEMBL + PubChem	Phase 4 (approved)	FGFR2
AXITINIB	ChEMBL	Phase 4 (approved)	FGFR2
BRIGATINIB	ChEMBL	Phase 4 (approved)	FGFR2
CERITINIB	ChEMBL	Phase 4 (approved)	FGFR2
DASATINIB	ChEMBL	Phase 4 (approved)	FGFR2
ERDAFITINIB	ChEMBL	Phase 4 (approved)	FGFR2
ERLOTINIB	ChEMBL	Phase 4 (approved)	FGFR2
FEDRATINIB	ChEMBL	Phase 4 (approved)	FGFR2
FUTIBATINIB	ChEMBL	Phase 4 (approved)	FGFR2
IBRUTINIB	ChEMBL	Phase 4 (approved)	FGFR2
INFIGRATINIB	ChEMBL	Phase 4 (approved)	FGFR2
LENVATINIB	ChEMBL	Phase 4 (approved)	FGFR2
MIDOSTAURIN	ChEMBL	Phase 4 (approved)	FGFR2
NINTEDANIB	ChEMBL	Phase 4 (approved)	FGFR2
NINTEDANIB ESYLATE	ChEMBL	Phase 4 (approved)	FGFR2
PEMIGATINIB	ChEMBL	Phase 4 (approved)	FGFR2
PONATINIB	ChEMBL	Phase 4 (approved)	FGFR2
SORAFENIB	ChEMBL	Phase 4 (approved)	FGFR2
SUNITINIB	ChEMBL	Phase 4 (approved)	FGFR2
VANDETANIB	ChEMBL	Phase 4 (approved)	FGFR2
BRIVANIB	ChEMBL	Phase 3	FGFR2
CEDIRANIB	ChEMBL	Phase 3	FGFR2
DOVITINIB	ChEMBL	Phase 3	FGFR2
LESTAURTINIB	ChEMBL	Phase 3	FGFR2
LINIFANIB	ChEMBL	Phase 3	FGFR2
SEMAXANIB	ChEMBL	Phase 3	FGFR2
AKN-028	ChEMBL	Phase 2	FGFR2
AT-9283	ChEMBL	Phase 2	FGFR2
BMS-754807	ChEMBL	Phase 2	FGFR2
DERAZANTINIB	ChEMBL	Phase 2	FGFR2
DORAMAPIMOD	ChEMBL	Phase 2	FGFR2
E-7090	ChEMBL	Phase 2	FGFR2
FEXAGRATINIB	ChEMBL	Phase 2	FGFR2
FGFR INHIBITOR DEBIO 1347	ChEMBL	Phase 2	FGFR2
FISOGATINIB	ChEMBL	Phase 2	FGFR2
FORETINIB	ChEMBL	Phase 2	FGFR2
LIRAFUGRATINIB	ChEMBL	Phase 2	FGFR2
LUCITANIB	ChEMBL	Phase 2	FGFR2
MK-2461	ChEMBL	Phase 2	FGFR2
OSI-632	ChEMBL	Phase 2	FGFR2
R-406	ChEMBL	Phase 2	FGFR2
REBASTINIB	ChEMBL	Phase 2	FGFR2
RESIGRATINIB	ChEMBL	Phase 2	FGFR2
RX-518	ChEMBL	Phase 2	FGFR2
SEGIGRATINIB	ChEMBL	Phase 2	FGFR2
SU-014813	ChEMBL	Phase 2	FGFR2
TANDUTINIB	ChEMBL	Phase 2	FGFR2
TOZASERTIB	ChEMBL	Phase 2	FGFR2
Afatinib	PubChem	Approved	FGFR2
Crizotinib	PubChem	Approved	FGFR2
Gefitinib	PubChem	Approved	FGFR2
Idelalisib	PubChem	Approved	FGFR2
Selumetinib	PubChem	Approved	FGFR2