Drug · Small Molecule

Mirdametinib

CI-DRUG-00000379Explore in graph →NCIt C52195 CHEMBL507361 CIViC Phase 2

Regulatory

Approvals (1)

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

emaRegulatory1 approval record · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
EU EMA	—	Ezmekly as monotherapy is indicated for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above.conditional	approved	Jul 17, 2025	ema

Data updated 14 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	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 2	9	8	20	1	EU	Jul 17, 2025	2004-02
Leukemia	Phase2	Phase 2	1	1	1	0	—	—	May 27, 2026
Non-Hodgkin Lymphoma	Phase2	Phase 2	1	1	2	0	—	—	May 27, 2026
Malignant Breast Neoplasm	Phase2	Phase 2	0	0	1	0	—	—

Curated evidence

Clinical evidence (11)

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

civicCuratedShowing 1–11 of 11 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
NF1 Mutation1
Mirdametinib + Sirolimus

Clinical trials

Trials with this intervention (21)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–21 of 21 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
NCT06876142	Combination Therapy (Mirdametinib and Sirolimus) for RAS Mutated Relapsed Refractory Multiple Myeloma	suspended	Phase 1 / Phase 2	54	National Cancer Institute (NCI)NIH	1	Sep 28, 2026	clinicaltrials
NCT06843967	A Study of Mirdametinib in Combination With Palbociclib in People With Liposarcoma	recruiting	Phase 1 / Phase 2

Molecule

Record

Kind: Small Molecule
Mechanism: Dual specificity mitogen-activated protein kinase kinase 1 inhibitor; Dual specificity mitogen-activated protein kinase kinase 2 inhibitor
Targets: 2 genes
Aliases: Ezmekly, Gomekli, MEK Inhibitor PD0325901, Mirdametinib, PD-0325901, PD0325901, Pd 03525901, PD 901

Data updated 22 hours agoSource updated unknown

Identifiers

Codes (2)

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

System	Code	Label
ATC (WHO)	L01EE	Antineoplastic agents
EMA product	EMEA/H/C/006460	Ezmekly

Regulatory status is jurisdiction-specific and time-bound. Nothing on this page is a treatment recommendation.

BRAF V600E4
Mirdametinib	Lung Non-Small Cell CarcinomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	—	submitted	EID2143 In a genetic screen of 87 lung cancer cell lines, one MEK-dependent cell line HCC364 contained a BRAF-V600E mutation. In growth assays, the MEK inhibitor PD-0325901 reduced proliferation in growth ass… (full text at CIViC) PMID 19010912 · Pratilas et al., 2008 · Open in CIViC	civic
〃	Melanoma	Predictive	D	Supports Sensitivity Response	—	submitted	EID2124 In a mouse in vivo study of MEK protein inhibitor, PD-0325901, was able to suppress growth of SKMEL28 BRAF-V600E xenograft tumors (P<0.01). The reduction of growth was associated with loss of D-cyclin… (full text at CIViC) PMID 16273091 · Solit et al., 2006 · Open in CIViC	civic
〃	Melanoma	Predictive	D	Supports Sensitivity Response	—	submitted	EID2125 An inducible BRAF-V600E mouse melanoma model shows a tight correlation between activated BRAF and disease progression. PMID 16424035 · Hoeflich et al., 2006 · Open in CIViC	civic
Mirdametinib + TrametinibSubstitutes	Melanoma	Predictive	D	Does Not Support Resistance	3	accepted	EID86 In the setting of BRAF(V600E), NF1 loss resulted in elevated activation of RAS-GTP but does not show resistance to MEK inhibitors. PMID 24576830 · Nissan et al., 2014 · Open in CIViC	civic

BRAF V600E4

Mirdametinib

Lung Non-Small Cell CarcinomaCURATED_BROADER

Predictive

Supports Sensitivity Response

—

submitted

EID2143

In a genetic screen of 87 lung cancer cell lines, one MEK-dependent cell line HCC364 contained a BRAF-V600E mutation. In growth assays, the MEK inhibitor PD-0325901 reduced proliferation in growth ass… (full text at CIViC)

PMID 19010912 · Pratilas et al., 2008 · Open in CIViC

civic

〃

Melanoma

Predictive

Supports Sensitivity Response

—

submitted

EID2124

In a mouse in vivo study of MEK protein inhibitor, PD-0325901, was able to suppress growth of SKMEL28 BRAF-V600E xenograft tumors (P<0.01). The reduction of growth was associated with loss of D-cyclin… (full text at CIViC)

PMID 16273091 · Solit et al., 2006 · Open in CIViC

civic

〃

Melanoma

Predictive

Supports Sensitivity Response

—

submitted

EID2125

An inducible BRAF-V600E mouse melanoma model shows a tight correlation between activated BRAF and disease progression.

PMID 16424035 · Hoeflich et al., 2006 · Open in CIViC

civic

Mirdametinib + TrametinibSubstitutes

Melanoma

Predictive

Does Not Support Resistance

accepted

EID86

In the setting of BRAF(V600E), NF1 loss resulted in elevated activation of RAS-GTP but does not show resistance to MEK inhibitors.

PMID 24576830 · Nissan et al., 2014 · Open in CIViC

civic

KRAS A146T1
Mirdametinib	Malignant Colorectal NeoplasmCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	submitted	EID2944 Preclinical data supporting resistance of Exon 4 mutations, in particular A146T, to cetuximab and sensitivity to PD0325901. PMID 20570890 · Janakiraman et al., 2010 · Open in CIViC	civic

KRAS A146T1

Mirdametinib

Malignant Colorectal NeoplasmCURATED_BROADER

Predictive

Supports Sensitivity Response

submitted

EID2944

Preclinical data supporting resistance of Exon 4 mutations, in particular A146T, to cetuximab and sensitivity to PD0325901.

PMID 20570890 · Janakiraman et al., 2010 · Open in CIViC

civic

HRAS G12V + HRAS Q61L + HRAS Q61R1
Binimetinib + Everolimus + Mirdametinib + MTOR Kinase Inhibitor AZD8055 + SelumetinibSubstitutes	Malignant NeoplasmALIAS	Predictive	D	Supports Sensitivity Response	3	accepted	EID700 In preclinical cancer cell line and Ba/F3 models, HRAS Q61L, Q61R, and G12V mutations conferred sensitivity to MEK inhibitors (AZD6244/selumetinib, MEK162/binimetinib, and PD0325901) and mTOR inhibito… (full text at CIViC) PMID 26544513 · Kiessling et al., 2015 · Open in CIViC	civic

HRAS G12V + HRAS Q61L + HRAS Q61R1

Binimetinib + Everolimus + Mirdametinib + MTOR Kinase Inhibitor AZD8055 + SelumetinibSubstitutes

Malignant NeoplasmALIAS

Predictive

Supports Sensitivity Response

accepted

EID700

In preclinical cancer cell line and Ba/F3 models, HRAS Q61L, Q61R, and G12V mutations conferred sensitivity to MEK inhibitors (AZD6244/selumetinib, MEK162/binimetinib, and PD0325901) and mTOR inhibito… (full text at CIViC)

PMID 26544513 · Kiessling et al., 2015 · Open in CIViC

civic

TCF19 EXPRESSION1
Birabresib + MirdametinibCombination	Melanoma	Predictive	D	Supports Sensitivity Response	2	submitted	EID9314 To understand the role of TCF19 expression in NRAS-mutant melanoma, an analysis of a TCGA cutaneous melanoma data set showed that patient survival was inversely correlated with levels of BRD4 and TCF1… (full text at CIViC) PMID 29650805 · Echevarría-Vargas et al., 2018 · Open in CIViC	civic

TCF19 EXPRESSION1

Birabresib + MirdametinibCombination

Melanoma

Predictive

Supports Sensitivity Response

submitted

EID9314

To understand the role of TCF19 expression in NRAS-mutant melanoma, an analysis of a TCGA cutaneous melanoma data set showed that patient survival was inversely correlated with levels of BRD4 and TCF1… (full text at CIViC)

PMID 29650805 · Echevarría-Vargas et al., 2018 · Open in CIViC

civic

DDX43 Overexpression1
Mirdametinib + Selumetinib + TrametinibSubstitutes	Uveal Melanoma	Predictive	D	Supports Resistance	4	accepted	EID1135 MEK inhibitor-resistant (selumetinib, PD0325901, GSK1129212) uveal melanoma cell lines were created from two GNAQ Q209L mutant cell lines. Overexpression of DDX43 mediated RAS-induced selumetinib resi… (full text at CIViC) PMID 24899684 · Ambrosini et al., 2014 · Open in CIViC	civic

DDX43 Overexpression1

Mirdametinib + Selumetinib + TrametinibSubstitutes

Uveal Melanoma

Predictive

Supports Resistance

accepted

EID1135

MEK inhibitor-resistant (selumetinib, PD0325901, GSK1129212) uveal melanoma cell lines were created from two GNAQ Q209L mutant cell lines. Overexpression of DDX43 mediated RAS-induced selumetinib resi… (full text at CIViC)

PMID 24899684 · Ambrosini et al., 2014 · Open in CIViC

civic

GNA11 Mutation1
Mirdametinib + Sotrastaurin AcetateCombination	Uveal Melanoma	Predictive	D	Supports Sensitivity Response	3	accepted	EID1952 Preclinical study in uveal melanoma. Increased PKC activity was found in uveal melanoma cell lines with GNA11 or GNAQ mutations (n=6) compared to GNA wt cell lines (n=4) and could be induced by introd… (full text at CIViC) PMID 24141786 · Chen et al., 2014 · Open in CIViC	civic

GNA11 Mutation1

Mirdametinib + Sotrastaurin AcetateCombination

Uveal Melanoma

Predictive

Supports Sensitivity Response

accepted

EID1952

Preclinical study in uveal melanoma. Increased PKC activity was found in uveal melanoma cell lines with GNA11 or GNAQ mutations (n=6) compared to GNA wt cell lines (n=4) and could be induced by introd… (full text at CIViC)

PMID 24141786 · Chen et al., 2014 · Open in CIViC

civic

GNAQ Mutation1
Mirdametinib + Sotrastaurin AcetateCombination	Uveal Melanoma	Predictive	D	Supports Sensitivity Response	4	accepted	EID1953 Preclinical study in uveal melanoma. Increased PKC activity was found in uveal melanoma cell lines with GNA11 or GNAQ mutations (n=6) compared to GNA wt cell lines (n=4) and could be induced by introd… (full text at CIViC) PMID 24141786 · Chen et al., 2014 · Open in CIViC	civic

GNAQ Mutation1

Mirdametinib + Sotrastaurin AcetateCombination

Uveal Melanoma

Predictive

Supports Sensitivity Response

accepted

EID1953

PMID 24141786 · Chen et al., 2014 · Open in CIViC

civic