TheraRadar
Data updated: May 26, 2026

KOSELUGO (selumetinib)

Mitogen-Activated Protein Kinase Kinase 1 Inhibitors Genetic Support Trial Activity: Stable 20 active trials
First-in-Class Orphan Drug Breakthrough Therapy Priority Review
Oncology Approved 2020-04-10

Koselugo is a kinase inhibitor used for adults and children aged one year and older who have neurofibromatosis type 1. It specifically helps patients with plexiform neurofibromas that cause symptoms and cannot be removed through surgery. This medication is prescribed to manage these complex nerve tumors when they are considered inoperable.

Source: FDA Label • AstraZeneca • Kinase Inhibitor
Jump to: Indications Approvals Trials Competitors Similar Safety Patents

How KOSELUGO Works

This drug works by blocking MEK1 and MEK2, which are proteins that regulate the ERK pathway within the body's RAS-regulated signaling system. By inhibiting these proteins, the medication reduces the phosphorylation of ERK, which helps decrease the number, volume, and growth of neurofibromas.

Source: FDA Label

Development Insights

AstraZeneca conducting 21 trials (30%)
164 indications explored (Broad Platform)
→ solid tumours (8 trials)
→ neurofibromatosis 1 (6 trials)
→ neurofibromatosis type 1 (4 trials)
4
Indications
--
Phase 3 Trials
2
Priority Reviews
6
Years on Market

Details

Status
Prescription
First Approved
2020-04-10
Patent Cliff
2029

Pro Metrics

Patent cliff and revenue data

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Routes
ORAL
Dosage Forms
CAPSULE, GRANULE

Companies

AstraZeneca
Active Ingredient: SELUMETINIB

KOSELUGO Approval History

2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
18 FDA actions from 2020 to 2025 · 1 indication expansions
Nov 2025 SUPPL
Label · Labeling
FDA Letter Label
Sep 2025 ORIGINAL Priority
New Form · Type 3 - New Dosage Form
FDA Letter Label
Jan 2024 SUPPL
Efficacy
FDA Letter Label

What KOSELUGO Treats

2 indications

KOSELUGO is approved for 2 conditions since its original approval in 2020. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Neurofibromatosis Type 1
  • Plexiform Neurofibroma
Source: FDA Label

KOSELUGO Target & Pathway

Pro

Target

MEK (Mitogen-Activated Protein Kinase Kinase) Serine/Threonine Kinase

A kinase downstream of BRAF in the MAPK pathway. MEK inhibitors are often combined with BRAF inhibitors to provide more complete pathway blockade and delay resistance. This combination is standard treatment for BRAF-mutant melanoma.

KOSELUGO Competitive Set

Pro

Three rings of competition based on shared molecular targets and treated indications.

Direct competitors

1

Same target(s) AND same indication — head-to-head.

GOMEKLI
SPRINGWORKS
2025 1 indic.

MoA expansion candidates

4

Same target(s), different indications — where else is this mechanism being explored?

AVMAPKI FAKZYNJA CO-PACK (COPACKAGED)
VERASTEM INC
2025 1 indic.
MEKTOVI
ARRAY BIOPHARMA
2018 2 indic.
Unlock 2 more competitors across all three rings.
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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 KOSELUGO

FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.

GOMEKLI
MIRDAMETINIB
1 shared
SPRINGWORKS
Shared indications:
Neurofibromatosis Type 1
📋

Clinical Trial Registry

66 trials
Trial Sponsor ID Phase Status Title
NCT01089101 NCI-2012-03173 NCI-2012-03173, PBTC-029B Ph 1, Ph 2 active not recruiting Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
NCT02664935 RG_14-072 2014-000814-73, ISRCTN38344105 Ph 2 completed National Lung Matrix Trial: Multi-drug Phase II Trial in Non-Small Cell Lung Cancer
NCT03162627 2016-1129 NCI-2018-01205 Ph 1 active not recruiting Selumetinib and Olaparib in Solid Tumors
NCT01364051 NCI-2012-02906 NCI-2012-02906, CDR0000700596 Ph 1 active not recruiting Cediranib Maleate and Selumetinib Sulfate in Treating Patients With Solid Malignancies
NCT04924608 KOMET results posted D134BC00001 2020-005607-39 Ph 3 active not recruiting Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
NCT06188741 NF114 IRB-300010135 164893, W81XWH-22-3-0001 Ph 2 recruiting Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
NCT02407405 160043 16-C-0043 Ph 2 active not recruiting MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
NCT03801369 results posted STUDY00018504 NCI-2019-00388, STUDY00018504 Ph 2 terminated AMTEC IIT: Phase 2 Multiarm Study in TNBC
NCT05101148 D1346C00015 2024-517216-29-00, 2020-005648-52 Ph 1 active not recruiting Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
NCT04590235 D1346C00011 Ph 1 active not recruiting A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
NCT03392246 17-540 Ph 2 active not recruiting A Phase 2 Study of Osimertinib in Combination With Selumetinib in EGFR Inhibitor naïve Advanced EGFR Mutant Lung Cancer
NCT02813135 ESMART 2016-000133-40 2016/2396, 2024-514791-40-00 Ph 1, Ph 2 recruiting European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors
NCT02546661 BISCAY D2615C00001 GU 118, BISCAY Ph 1 active not recruiting Open-Label, Randomised, Multi-Drug, Biomarker-Directed, Phase 1b Study in Pts w/ Muscle Invasive Bladder Cancer
NCT03326310 IRB17-0774 Ph 1 recruiting Selumetinib and Azacitidine in High Risk Chronic Blood Cancers
NCT02151084 BIL-MEK Ph 2 active not recruiting A Study of Different Dosing Schedules of Selumetinib With Cisplatin/Gemcitabine (CIS/GEM) Versus CIS/GEM Alone in Biliary Cancer
NCT01933932 SELECT-1 results posted D1532C00079 2013-001676-38 Ph 3 active not recruiting Assess Efficacy & Safety of Selumetinib in Combination With Docetaxel in Patients Receiving 2nd Line Treatment for v-Ki-ras2 Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) Positive NSCLC
NCT03581487 2017-0888 NCI-2018-01098, 2017-0888 Ph 1, Ph 2 completed Durvalumab, Tremelimumab, and Selumetinib in Treating Participants With Recurrent or Stage IV Non-small Cell Lung Cancer
NCT06735820 MEKMDM2 STUDY00001200 W81XWH-22-1-1120 Ph 1, Ph 2 not yet recruiting Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST
NCT03095248 SEL-TH-1601 results posted SEL-TH-1601 2016-8833 Ph 2 terminated Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors
NCT02188264 NCI-2014-01484 NCI-2014-01484, 13-2628 Ph 1 completed Selumetinib and Cyclosporine in Treating Patients With Advanced Solid Tumors or Advanced or Metastatic Colorectal Cancer
NCT03213691 results posted NCI-2017-01250 NCI-2017-01250, APEC1621E Ph 2 completed Selumetinib Sulfate in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
NCT03944772 ORCHARD D6186C00001 2023-504624-25-00, 2018-003974-29 Ph 2 active not recruiting Phase 2 Platform Study in Patients With Advanced Non-Small Lung Cancer Who Progressed on First-Line Osimertinib Therapy (ORCHARD)
NCT02299999 SAFIR02_Breast UC-0105/1304 2013-001652-36 Ph 2 active not recruiting SAFIR02_Breast - Efficacy of Genome Analysis as a Therapeutic Decision Tool for Patients With Metastatic Breast Cancer
NCT06763315 LS-NF1PNs SEL20241219 Ph 2 not yet recruiting Low-dose Selumetinib for the Treatment of Plexiform Neurofibromas in Chinese Children
NCT03833427 results posted 5618-001 MK-5618-001 Ph 1 terminated Study of Selumetinib (MK-5618) in Combination With Pembrolizumab (MK-3475) in Participants With Advanced/Metastatic Solid Tumors (MK-5618-001)
NCT06621082 SYSKY-2024-284-02 Ph 2 not yet recruiting The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate Capsule
NCT06620354 SYSKY-2024-285-02 Ph 2 not yet recruiting Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule
NCT02393690 results posted RU241306I NCI-2015-00277, 14-008494 Ph 2 completed Iodine I-131 With or Without Selumetinib in Treating Patients With Recurrent or Metastatic Thyroid Cancer
NCT03326388 INSPECT 15Hi53 Ph 1, Ph 2 completed Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours
NCT02337530 results posted I219 Ph 2 completed Selumetinib in Patients Receiving Pemetrexed and Platinum-based Chemotherapy in Advanced or Metastatic KRAS Wildtype or Unknown Non-Squamous NSCLC
NCT02117167 SAFIR02_Lung UC-0105/1305 / IFCT 1301 2013-001653-27 Ph 2 completed SAFIR02_Lung - Efficacy of Targeted Drugs Guided by Genomic Profiles in Metastatic NSCLC Patients
NCT03433183 SARC031 CDMRP-NF150092 Ph 2 completed SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
NCT05825365 PROTECT D1346C00018 Ph 2 withdrawn Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study
NCT01783197 I215 Ph 1 completed Study of Selumetinib in Patients With Previously Treated or Untreated Advanced/Metastatic NSCLC
NCT03745989 results posted 8353-014 MK-8353-014 Ph 1 completed Study of MK-8353 + Selumetinib in Advanced/Metastatic Solid Tumors (MK-8353-014)
NCT03705507 SeluDex RG_16-186 2016-003904-29, ISRCTN92323261 Ph 1, Ph 2 terminated International Trial of Selumetinib in Combination With Dexamethasone for the Treatment of Acute Lymphoblastic Leukaemia
NCT04495127 D1346C00013 Ph 1 completed Selumetinib Paediatric NF1 Japan Study
NCT02503358 IRB00011462 NCI-2015-01018, 11,462 Ph 1 completed Selumetinib and Paclitaxel as Second-Line Treatment in Treating Patients With Stage IIIB-IV Non-small Cell Lung Cancer
NCT01222689 results posted NCI-2011-01266 NCI-2011-01266, CDR0000686634 Ph 2 completed Selumetinib and Erlotinib Hydrochloride in Treating Patients With Locally Advanced or Metastatic Pancreatic Cancer
NCT02586987 D1345C00003 Ph 1 completed A Study to Assess the Safety, Tolerability and Anti-tumour Activity of Ascending Doses of Selumetinib in Combination With MEDI4736 and Selumetinib in Combination With MEDI4736 and Tremelimumab in Patients With Advanced Solid Tumours
NCT01843062 ASTRA results posted D1532C00065 2013-000423-14 Ph 3 terminated Comparing Complete Remission After Treatment With Selumetinib/Placebo in Patient With Differentiated Thyroid Cancer
NCT01011933 results posted NCI-2011-01958 NCI-2011-01958, GOG-0229H Ph 2 completed Selumetinib in Treating Patients With Recurrent or Persistent Endometrial Cancer
NCT01160718 SAKK 21/08 SWS-SAKK-21/08, EUDRACT-2010-019965-27 Ph 2 completed Fulvestrant With or Without AZD6244 in Treating Patients With Advanced Breast Cancer That Progressed After Aromatase Inhibitor Therapy
NCT01752569 SCART STH16059 2011-003099-35 Ph 1, Ph 2 terminated A Study of Selumetinib in Patients With Kaposi's Sarcoma
NCT02025114 201312070MIPC Ph 1, Ph 2 completed Selumetinib in Combination With Gefitinib in NSCLC Patients
NCT03004105 2016-0060 Ph 2 withdrawn MEDI4736 With Selumetinib for KRAS Mutant Non-Small Cell Lung Cancer (NSCLC)
NCT01809210 SELECT-3 results posted D1532C00070 EudraCT number: 2012-005202-22 Ph 1 completed Assess Safety & Efficacy of Selumetinib When Given in Combination With Standard First Line Treatment for Advanced Non-small Cell Lung Cancer
NCT01143402 results posted NCI-2011-01411 NCI-2011-01411, CDR0000674866 Ph 2 completed Temozolomide or Selumetinib in Treating Patients With Metastatic Melanoma of the Eye
NCT01242605 ABC-04 UCL/10/0254 2010-018522-39 Ph 1 completed ABC-04 a Study of Cisplatin, Gemcitabine and Selumetinib in Patients With Advanced Biliary Tract Cancer
NCT01949870 results posted D1532C00075 Ph 1 terminated Selumetinib (AZD6244, ARRY-142886) J-BTC Phase 1 Study

Showing 50 of 66 trials

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Active Pipeline

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Ongoing clinical trials by development phase

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

ClinicalTrials.gov ↗ Pro

Full development history with FDA approval milestones

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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.

KOSELUGO FDA Label Details

Indications & Usage

FDA Label (PDF)

KOSELUGO is indicated for the treatment of Neurofibromatosis Type 1; Plexiform Neurofibroma.

View full patent landscape →
8 OB patents · 3 families · 263 international docs across 46 countries

KOSELUGO Patents & Exclusivity

Latest Patent: Mar 2029
Exclusivity: Nov 2028

Patents (8 active)

US12364684 Expires Mar 26, 2029
US11813246 Expires Mar 26, 2029
US12318367 Expires Mar 26, 2029
US12220403 Expires Mar 26, 2029
US7425637 Expires Mar 13, 2028
US9562017 Expires Dec 12, 2026
US9156795 Expires Dec 12, 2026
US8178693 Expires Mar 13, 2026

Exclusivity

NP Until Sep 2028
NPP Until Nov 2028
ODE* Until Apr 2027
NP Until Sep 2028
NPP Until Nov 2028
ODE* Until Apr 2027
NP Until Sep 2028
NPP Until Nov 2028
ODE* Until Apr 2027
NP Until Sep 2028
NPP Until Nov 2028
ODE* Until Apr 2027
NP Until Sep 2028
NPP Until Nov 2028
ODE* Until Apr 2027
NP Until Sep 2028
NPP Until Nov 2028
ODE* Until Apr 2027
NP Until Sep 2028
NPP Until Nov 2028
ODE* Until Apr 2027
NP Until Sep 2028
NPP Until Nov 2028
ODE* Until Apr 2027
NPP Until Nov 2028
NPP Until Sep 2028
ODE-288 Until Apr 2027
NPP Until Sep 2028
NPP Until Nov 2028
ODE-288 Until Apr 2027
NPP Until Nov 2028
NPP Until Sep 2028
ODE-288 Until Apr 2027
NPP Until Sep 2028
NPP Until Nov 2028
ODE-288 Until Apr 2027
Source: FDA Orange Book

Pro Intelligence Preview

Deep insights for KOSELUGO

Revenue Insights

  • • Quarterly revenue tracking
  • • Historical trend analysis

Patent Timeline

  • • Cliff: 2029
  • • 64 active patents

Trial Analysis

  • • 70 total trials
  • • Stage: Stable

Competitive Landscape

  • • 1 similar drugs
  • • Same target/indication analysis
Unlock Full Intelligence

Full approval history • All patents • Revenue trends • Competitor analysis

Data Sources

FDA Approval: NDA219943 → Label: DailyMed → Patents: FDA Orange Book → Trials: ClinicalTrials.gov →

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