Mirdametinib

Last updated

Mirdametinib
Mirdametinib.svg
Clinical data
Trade names Gomekli
Other namesPD-0325901
AHFS/Drugs.com Monograph
MedlinePlus a625043
License data
Routes of
administration 		By mouth
Drug class Antineoplastic
ATC code
Legal status
Legal status
Identifiers
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
PDB ligand
CompTox Dashboard (EPA)
ECHA InfoCard 100.213.070 OOjs UI icon edit-ltr-progressive.svg
Chemical and physical data
Formula C16H14F3IN2O4
Molar mass 482.198 g·mol−1
3D model (JSmol)
  • O=C(NOCC(O)CO)C1=CC=C(F)C(F)=C1NC2=CC=C(I)C=C2F
  • InChI=1S/C16H14F3IN2O4/c17-11-3-2-10(16(25)22-26-7-9(24)6-23)15(14(11)19)21-13-4-1-8(20)5-12(13)18/h1-5,9,21,23-24H,6-7H2,(H,22,25)/t9-/m1/s1
  • Key:SUDAHWBOROXANE-SECBINFHSA-N

Mirdametinib, sold under the brand name Gomekli, is a medication used for the treatment of people with neurofibromatosis type 1. [1] Mirdametinib is a kinase inhibitor. [1] [4] It is taken by mouth. [1]

Contents

The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. [5] The most common grade 3 or 4 laboratory abnormalities include increased creatine phosphokinase. [5] The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea. [5] The most common grade 3 or 4 laboratory abnormalities include decreased neutrophil count and increased creatine phosphokinase. [5]

Mirdametinib was approved for medical use in the United States in February 2025. [1] [5]

Medical uses

Mirdametinib is indicated for the treatment of people with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection. [1]

Adverse effects

The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. [5] The most common grade 3 or 4 laboratory abnormalities include increased creatine phosphokinase. [5] The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea. [5] The most common grade 3 or 4 laboratory abnormalities include decreased neutrophil count and increased creatine phosphokinase. [5]

Mirdametinib can cause left ventricular dysfunction and ocular toxicity including retinal vein occlusion, retinal pigment epithelial detachment, and blurred vision. [5]

History

The efficacy of mirdametinib was evaluated in ReNeu (NCT03962543), a multicenter, single-arm trial in 114 participants aged two years of age and older (58 adults, 56 pediatric participants) with symptomatic, inoperable NF1-associated plexiform neurofibromas causing significant morbidity. [5] An inoperable plexiform neurofibromas was defined as a plexiform neurofibromas that could not be completely surgically removed without risk for substantial morbidity due to encasement or close proximity to vital structures, invasiveness, or high vascularity. [5]

The US Food and Drug Administration (FDA) granted the application for mirdametinib priority review, fast track, and orphan drug designations along with a priority review voucher. [5]

Society and culture

Mirdametinib was approved for medical use in the United States in February 2025. [5] [6] [7]

In May 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Ezmekly, intended for the treatment of plexiform neurofibromas (PN) in adults and children from two years of age with neurofibromatosis type 1 (NF1). [2] The applicant for this medicinal product is SpringWorks Therapeutics Ireland Limited. [2] Mirdametinib was authorized for medical use in the EU in July 2025. [3]

References

  1. 1 2 3 4 5 6 "Gomekli- mirdametinib capsule; Gomekli- mirdametinib tablet, for suspension". DailyMed. 27 February 2025. Retrieved 2 April 2025.
  2. 1 2 3 "Ezmekly EPAR". European Medicines Agency (EMA). 23 May 2025. Retrieved 15 June 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  3. 1 2 "Ezmekly PI". Union Register of medicinal products. 18 July 2025. Retrieved 31 July 2025.
  4. ↑ Armstrong AE, Belzberg AJ, Crawford JR, Hirbe AC, Wang ZJ (June 2023). "Treatment decisions and the use of MEK inhibitors for children with neurofibromatosis type 1-related plexiform neurofibromas". BMC Cancer. 23 (1) 553. doi: 10.1186/s12885-023-10996-y . PMC   10273716 . PMID   37328781.
  5. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 "FDA approves mirdametinib for adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection". U.S. Food and Drug Administration (FDA). 11 February 2025. Archived from the original on 13 February 2025. Retrieved 16 February 2025.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  6. ↑ "UPDATE: SpringWorks Therapeutics Announces FDA Approval of Gomekli (mirdametinib) for the Treatment of Adult and Pediatric Patients with NF1-PN" (Press release). SpringWorks Therapeutics. 12 February 2025. Archived from the original on 13 February 2025. Retrieved 16 February 2025– via GlobeNewswire News Room.
  7. ↑ "Novel Drug Approvals for 2025". U.S. Food and Drug Administration (FDA). 14 February 2025. Archived from the original on 3 March 2025. Retrieved 16 February 2025.
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