Drugs / Mirdametinib
last change Aug 2026 re-read 2 minutes ago

Mirdametinib

also known as PD-0325901 · Pd 03525901 · Pd 901

Small molecule targets MAP2K1, MAP2K2 via inhibition

Developed for
neurofibromatosis type 1 · colorectal cancer · non-small cell lung carcinoma · breast neoplasm · cancer · cancer of unknown primary site · central nervous system neoplasm · childhood low-grade glioma
+18 more · chronic lymphocytic leukemia/small lymphocytic lymphoma · colonic neoplasm · dedifferentiated liposarcoma · glioma · Her2-receptor negative breast cancer · juvenile xanthogranuloma · Langerhans cell histiocytosis · low grade glioma · lung cancer · malignant peripheral nerve sheath tumor · melanoma · metastatic melanoma · neoplasm · neurofibroma · plexiform neurofibroma · refractory plasma cell neoplasm · sinus histiocytosis with massive lymphadenopathy · vascular malformation
Investigated by
SpringWorks Therapeutics, Inc. · Pfizer · Memorial Sloan Kettering Cancer Center · SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany · National Cancer Institute (NCI) · University of Alabama at Birmingham
+30 more · Array BioPharma · Beaumont Hospital · BeiGene · Belfast Health and Social Care Trust · Centre Georges Francois Leclerc · Children's Hospital Medical Center, Cincinnati · Children's Hospital of Philadelphia · Congressionally Directed Medical Research Programs · Dana-Farber Cancer Institute · European Commission · European Georges Pompidou Hospital · Hospital Vall d'Hebron · Johns Hopkins University · Kevin Kim, MD · Murdoch Childrens Research Institute · Oxford University Hospitals NHS Trust · Peter MacCallum Cancer Centre, Australia · Queen's University, Belfast · Royal Children's Hospital · Saint Antoine University Hospital · Sharp HealthCare · St. Jude Children's Research Hospital · St. Justine's Hospital · The Netherlands Cancer Institute · University Hospital, Antwerp · University of Minnesota · University of Oxford · University of Paris 5 - Rene Descartes · University of Turin, Italy · Velindre NHS Trust

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved · conditional EU (EMA) Ezmekly 2025-07-17 europa.eu
Approved US (FDA) GOMEKLI 2025-02-11 fda.gov

Trials 27 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT06693284 Nov 2025 → Jan 2027 expected malignant peripheral nerve sheath tumor University of Minnesota Recruiting No outcome recorded
Phase 16 trials
Phase 1 NCT07279233 Dec 2025 → Feb 2026 SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany Completed No outcome recorded
Phase 1 NCT06997276 May 2025 → Sep 2026 expected SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany Recruiting No outcome recorded
Phase 1 NCT03905148 May 2019 → Oct 2024 cancer BeiGene Completed No outcome recorded
Phase 1 NCT02510001 Nov 2014 → Dec 2018 colorectal cancer University of Oxford Completed No outcome recorded
Phase 1 NCT02022982 Jan 2014 → Nov 2017 lung cancer Dana-Farber Cancer Institute Completed No outcome recorded
Phase 1 NCT01347866 Oct 2011 → Dec 2015 cancer of unknown primary site Pfizer Terminated No outcome recorded
Phase 1/211 trials
Phase 1/2 NCT06876142 Jul 2026 → Jan 2033 plasma cell myeloma, refractory plasma cell neoplasm National Cancer Institute (NCI) Suspended No outcome recorded
Phase 1/2 NCT05937906 Jul 2026 → Jul 2028 expected non-small cell lung carcinoma Centre Georges Francois Leclerc Recruiting No outcome recorded
Phase 1/2 NCT07539441 Apr 2026 → Oct 2029 expected central nervous system neoplasm, glioma Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 1/2 NCT06666348 Jan 2026 → Apr 2030 expected childhood low-grade glioma St. Justine's Hospital Recruiting No outcome recorded
Phase 1/2 NCT06843967 Feb 2025 → Aug 2028 expected dedifferentiated liposarcoma, myxoid liposarcoma, pleomorphic liposarcoma, round cell liposarcoma +1 Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 1/2 NCT06159166 Feb 2024 → Nov 2027 expected neurofibroma, neurofibromatosis type 1 Johns Hopkins University Recruiting No outcome recorded
Phase 1/2 NCT05580770 Feb 2023 → Jan 2025 neoplasm SpringWorks Therapeutics, Inc. Terminated No outcome recorded
Phase 1/2 NCT05054374 Sep 2021 → Oct 2023 Her2-receptor negative breast cancer Memorial Sloan Kettering Cancer Center Completed No outcome recorded
Phase 1/2 NCT04923126 Jun 2021 → Jun 2031 expected low grade glioma St. Jude Children's Research Hospital Recruiting No outcome recorded
Phase 1/2 NCT02039336 Apr 2014 → Aug 2018 colorectal cancer The Netherlands Cancer Institute Completed No outcome recorded
Phase 28 trials
Phase 2 NCT07521657 Aug 2026 → Aug 2030 expected neurofibromatosis type 1 University of Alabama at Birmingham Not yet recruiting No outcome recorded
Phase 2 NCT07061951 May 2026 → Feb 2030 expected chronic lymphocytic leukemia/small lymphocytic lymphoma National Cancer Institute (NCI) Recruiting No outcome recorded
Phase 2 NCT07237100 Oct 2025 → Oct 2027 expected metastatic melanoma Kevin Kim, MD Recruiting No outcome recorded
Phase 2 NCT05983159 Sep 2024 → Jan 2027 expected PIK3CA-related overgrowth spectrum, arteriovenous hemangioma/malformation, cystic hygroma, lymphatic malformation +1 Murdoch Childrens Research Institute Recruiting No outcome recorded
Phase 2 NCT06153173 Feb 2024 → Mar 2030 expected Langerhans cell histiocytosis, juvenile xanthogranuloma, sinus histiocytosis with massive lymphadenopathy Children's Hospital Medical Center, Cincinnati Recruiting No outcome recorded
Phase 2 NCT03962543 Sep 2019 → Sep 2023 overdue neurofibromatosis type 1, plexiform neurofibroma SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany Active not recruiting No outcome recorded
Phase 2 NCT02096471 Jun 2014 → Aug 2017 neurofibromatosis type 1 University of Alabama at Birmingham Completed No outcome recorded
Phase 2 NCT00174369 Nov 2005 → Jun 2007 non-small cell lung carcinoma Pfizer Terminated No outcome recorded
Phase not stated1 trial
NCT02297802 Jun → Jul 2013 Sharp HealthCare No longer available No outcome recorded
Also used as a comparator or background therapy in 1 trial
PhaseRegistry idDatesIndicationSponsorStatusOutcome
NCT05028166 Background histiocytic and dendritic cell neoplasm, plexiform neurofibroma SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany Available No outcome recorded

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 30 sources stand behind the page.

FieldValueCited text
Known as Mirdametinib “NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas.” PMID 33507822 Jan 2021
20

NCT06159166

NCT07061951

NCT06693284

NCT06666348

NCT05028166

NCT07237100

NCT06843967

NCT05983159

NCT03905148

NCT06876142

NCT05054374

NCT07539441

NCT06997276

NCT05937906

NCT07279233

NCT04923126

NCT07521657

NCT03962543

NCT06153173

NCT05580770

Known as Pd 03525901 ChEMBL registry synonym — accepted as the source's own label CHEMBL507361
Known as Pd 901 ChEMBL registry synonym — accepted as the source's own label CHEMBL507361
Known as PD-0325901 “NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas.” PMID 33507822 Jan 2021
9

“This is a 2-part Phase 1b study of BGB-283 (lifirafenib) and PD-0325901 (mirdametinib) combination in participants with tumors.” NCT03905148

“Mirdametinib (PD-0325901) is a highly selective non-ATP-competitive inhibitor of MEK1 and MEK2.” NCT06843967

NCT01347866

NCT02022982

NCT02510001

NCT00174369

NCT02039336

NCT00147550

NCT02297802

Action Inhibit “Dual specificity mitogen-activated protein kinase kinase 2 inhibitor” CHEMBL507361
2

“Dual specificity mitogen-activated protein kinase kinase 1 inhibitor” CHEMBL507361

“PD-0325901 (oral MEK inhibitor)” NCT01347866

Modality Small molecule “highly selective small-molecule inhibitor” NCT03962543
Route Oral “PD-0325901 was administered orally at 20 mg twice daily (BID) for 21 consecutive days followed by 7 days of no treatment.” PMID 21516509 Apr 2011
Target MAP2K1 “Dual specificity mitogen-activated protein kinase kinase 1 inhibitor” CHEMBL507361
Target MAP2K2 “Dual specificity mitogen-activated protein kinase kinase 2 inhibitor” CHEMBL507361