GOMEKLI (mirdametinib)
Gomekli is a kinase inhibitor used for adults and children two years of age and older who have neurofibromatosis type 1. It helps patients with symptomatic plexiform neurofibromas that cannot be completely removed through surgery. This medication provides a therapeutic option for managing these specific nerve-related tumors when resection is not an option.
How GOMEKLI Works
This drug works by inhibiting MEK1 and MEK2, which are proteins that regulate the ERK signaling pathway. By blocking these kinases, the medication reduces the activity of downstream proteins and helps decrease the growth and volume of neurofibroma tumors.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2025-02-11
- Patent Cliff
- 2044
- Routes
- ORAL
- Dosage Forms
- CAPSULE, TABLET, FOR SUSPENSION
GOMEKLI Approval History
What GOMEKLI Treats
1 indicationsGOMEKLI is approved for 1 conditions since its original approval in 2025. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Neurofibromatosis Type 1
GOMEKLI Target & Pathway
ProTarget
GOMEKLI Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to GOMEKLI
FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
14 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT07539441 | 26-103 | Ph 1, Ph 2 | recruiting | A Study of Mirdametinib in People With Central Nervous System Tumors |
| NCT06876142 | 10001811 001811-C | Ph 1, Ph 2 | suspended | Combination Therapy (Mirdametinib and Sirolimus) for RAS Mutated Relapsed Refractory Multiple Myeloma |
| NCT07061951 | NCI-2025-04637 NCI-2025-04637, 10708 | Ph 2 | recruiting | Testing the Effectiveness of the Anti-cancer Drug, Mirdametinib, in Treating Relapsed, Refractory Chronic Lymphocytic Leukemia |
| NCT07279233 | MEK-NF1-105 | Ph 1 | completed | To Assess the Enzyme Inducing Effects of Carbamazepine on the PK of Mirdametinib in Healthy Participants |
| NCT05983159 TARGET-VM | 85218 | Ph 2 | recruiting | A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations |
| NCT06159166 Mirda | IRB00365255 J23129 | Ph 1, Ph 2 | recruiting | Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF). |
| NCT07521657 NF118 | IRB-300016184 | Ph 2 | not yet recruiting | Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma |
| NCT06843967 | 24-344 | Ph 1, Ph 2 | recruiting | A Study of Mirdametinib in Combination With Palbociclib in People With Liposarcoma |
| NCT06666348 MIRV | MIRV | Ph 1, Ph 2 | recruiting | Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK |
| NCT04923126 | SJ901 NCI-2021-05912 | Ph 1, Ph 2 | recruiting | SJ901: Evaluation of Mirdametinib in Children, Adolescents, and Young Adults With Low-Grade Glioma |
| NCT03905148 | BGB-283/PD-0325901-AU-001 | Ph 1 | completed | Study of the Safety and Pharmacokinetics of BGB-283 (Lifirafenib) and PD-0325901 (Mirdametinib) in Participants With Advanced or Refractory Solid Tumors |
| NCT07237100 | CPMC24-MEL01 | Ph 2 | recruiting | Mirdametinib in Patients With Advanced NF1-mutant Melanoma |
| NCT06153173 | 2021-0206 | Ph 2 | recruiting | Mirdametinib in Histiocytic Disorders |
| NCT05054374 results posted | 21-288 | Ph 1, Ph 2 | completed | A Study of Mirdametinib on Its Own or in Combination With Fulvestrant in People With Solid Tumor Cancer |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
GOMEKLI FDA Label Details
Indications & Usage
FDA Label (PDF)GOMEKLI is indicated for the treatment of Neurofibromatosis Type 1.
GOMEKLI Patents & Exclusivity
Patents (22 active)
Exclusivity
Pro Intelligence Preview
Deep insights for GOMEKLI
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2044
- • 94 active patents
Trial Analysis
- • 14 total trials
- • Stage: Growth
Competitive Landscape
- • 1 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment