CLINICAL TRIALS PROFILE FOR MEKTOVI

All Clinical Trials for MEKTOVI

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02465060 ↗	Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial)	Recruiting	National Cancer Institute (NCI)	Phase 2	2015-08-12	This phase II MATCH trial studies how well treatment that is directed by genetic testing works in patients with solid tumors or lymphomas that have progressed following at least one line of standard treatment or for which no agreed upon treatment approach exists. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with genetic abnormalities (such as mutations, amplifications, or translocations) may benefit more from treatment which targets their tumor's particular genetic abnormality. Identifying these genetic abnormalities first may help doctors plan better treatment for patients with solid tumors, lymphomas, or multiple myeloma.
NCT02902042 ↗	Encorafenib + Binimetinib + Pembrolizumab in Patients With Unresectable or Metastatic BRAF V600 Mutant Melanoma	Completed	Prof. Dr. med. Dirk Schadendorf	Phase 1/Phase 2	2018-04-24	This study will investigate the influence of maintenance therapy on progression-free survival (PFS) and overall survival (OS) after combination therapy with BRAF/MEK (MAP-ERK kinase) inhibitors and PD-1 antibody pembrolizumab. In the safety phase I part the optimal dose of pembrolizumab in combination with BRAF inhibitor and MEK inhibitor and the safety of this three-drugs-combination regime will be determined. In the randomized part 2 different maintenance therapies will be tested for toxicity and efficacy. Patients with disease control after 6 months of triple therapy will be randomized to receive 2 different maintenance therapies further on, either continuation of triple therapy or administration of pembrolizumab alone.
NCT02910700 ↗	Nivolumab With Trametinib and Dabrafenib, or Encorafenib and Binimetinib in Treating Patients With BRAF Mutated Metastatic or Unresectable Stage III-IV Melanoma	Recruiting	Bristol-Myers Squibb	Phase 2	2016-12-09	This phase II trial studies the side effects and how well nivolumab with trametinib and dabrafenib, or encorafenib and binimetinib work in treating patients with BRAF-mutated stage III-IV melanoma that has spread to other places in the body (metastatic) or cannot be removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as nivolumab, may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Trametinib, dabrafenib, encorafenib, and binimetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known if nivolumab with trametinib and dabrafenib, or encorafenib and binimetinib may work better in treating patients with BRAF-mutated melanoma.
NCT02910700 ↗	Nivolumab With Trametinib and Dabrafenib, or Encorafenib and Binimetinib in Treating Patients With BRAF Mutated Metastatic or Unresectable Stage III-IV Melanoma	Recruiting	National Cancer Institute (NCI)	Phase 2	2016-12-09	This phase II trial studies the side effects and how well nivolumab with trametinib and dabrafenib, or encorafenib and binimetinib work in treating patients with BRAF-mutated stage III-IV melanoma that has spread to other places in the body (metastatic) or cannot be removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as nivolumab, may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Trametinib, dabrafenib, encorafenib, and binimetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known if nivolumab with trametinib and dabrafenib, or encorafenib and binimetinib may work better in treating patients with BRAF-mutated melanoma.
NCT02910700 ↗	Nivolumab With Trametinib and Dabrafenib, or Encorafenib and Binimetinib in Treating Patients With BRAF Mutated Metastatic or Unresectable Stage III-IV Melanoma	Recruiting	M.D. Anderson Cancer Center	Phase 2	2016-12-09	This phase II trial studies the side effects and how well nivolumab with trametinib and dabrafenib, or encorafenib and binimetinib work in treating patients with BRAF-mutated stage III-IV melanoma that has spread to other places in the body (metastatic) or cannot be removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as nivolumab, may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Trametinib, dabrafenib, encorafenib, and binimetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known if nivolumab with trametinib and dabrafenib, or encorafenib and binimetinib may work better in treating patients with BRAF-mutated melanoma.
NCT03106415 ↗	Pembrolizumab and Binimetinib in Treating Patients With Locally Advanced or Metastatic Triple Negative Breast Cancer	Recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2017-09-26	This phase I/II trial studies the best dose of pembrolizumab and binimetinib and how well it works when given together with pembrolizumab in treating patients with triple negative breast cancer that has spread to other parts of the body (metastatic). Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Binimetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving pembrolizumab and binimetinib may work better in treating patients with triple negative breast cancer.

Clinical Trial Conditions for MEKTOVI

Condition Name

Condition Name for MEKTOVI
Intervention	Trials
Melanoma	7
Metastatic Melanoma	7
Advanced Malignant Solid Neoplasm	4
Malignant Melanoma	3

Condition MeSH

Condition MeSH for MEKTOVI
Intervention	Trials
Melanoma	18
Neoplasms	8
Carcinoma	7
Skin Neoplasms	6

Clinical Trial Locations for MEKTOVI

Trials by Country

Trials by Country for MEKTOVI
Location	Trials
United States	149
Germany	19
Spain	13
Italy	7
United Kingdom	6

Trials by US State

Trials by US State for MEKTOVI
Location	Trials
Texas	11
California	10
Florida	8
Massachusetts	8
Pennsylvania	6

Clinical Trial Progress for MEKTOVI

Clinical Trial Phase

Clinical Trial Phase for MEKTOVI
Clinical Trial Phase	Trials
Phase 3	2
Phase 2	19
Phase 1/Phase 2	7

Clinical Trial Status

Clinical Trial Status for MEKTOVI
Clinical Trial Phase	Trials
Recruiting	17
Not yet recruiting	12
Active, not recruiting	4

Clinical Trial Sponsors for MEKTOVI

Sponsor Name

Sponsor Name for MEKTOVI
Sponsor	Trials
National Cancer Institute (NCI)	16
Pierre Fabre Medicament	5
Pfizer	5

Sponsor Type

Sponsor Type for MEKTOVI
Sponsor	Trials
Other	33
Industry	29
NIH	16

Clinical Trials Update, Market Analysis, and Projection for Mektovi

Last updated: October 29, 2025

Introduction

Mektovi (selumetinib) is an oral MEK1/2 inhibitor developed by AstraZeneca, primarily approved for the treatment of neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas in pediatric patients aged 2 years and older. Since its initial approval, Mektovi has garnered attention in oncology and genetic disorder markets, with ongoing clinical trials expanding its indications. This report provides a comprehensive update on clinical trials, analyzes current market dynamics, and projects future growth opportunities for Mektovi.

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Clinical Trials Update

Current Phase and Indications

Mektovi’s clinical development has centered on Merlin pathway inhibition, targeting mutations in the RAS/MAPK pathway observed in various cancers and neurofibromatosis.

• Approved Indication: In 2020, the FDA approved Mektovi for pediatric patients aged 2 and older with symptomatic, inoperable plexiform neurofibromas associated with NF1 based on the phase 2 SPRINT trial [1].

• Ongoing Trials: Several pivotal studies are underway to evaluate the drug’s efficacy and safety in broader indications:

  • NCT03433179: A Phase 2 trial evaluating Mektovi in pediatric and adult patients with NF1-associated tumors (including plexiform neurofibromas and other tumors). Recruitment remains active, with preliminary data indicating tolerability and some tumor regression.

  • NCT04664936: A study assessing combined Mektovi with other pathway inhibitors for synergistic effect in NF1-related tumors.

  • NCT04383587: A trial exploring Mektovi’s role in treating advanced RAS/MAPK pathway-mutant tumors beyond NF1, including low-grade gliomas and other neurofibromas.

Trial Outcomes and Safety Profile

Early data affirm Mektovi's tolerability in pediatric and adult populations:

• Efficacy: Approximately 50-60% of patients with NF1-associated plexiform neurofibromas exhibited notable reduction (>20%) in tumor volume, with some cases showing significant regression [1].

• Safety: The most common adverse effects include fatigue, rash, diarrhea, and transient elevation in liver enzymes. Serious adverse events are rare but monitored closely.

• Biomarker Insights: Mutational analyses suggest that patients with NF1 mutations exhibit the most robust responses, aligning with Mektovi's mechanism centered on MEK inhibition.

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Market Analysis

Current Market Landscape

Mektovi occupies a unique niche in the targeted therapy landscape for neurofibromatosis and RAS/MAPK-driven cancers. Key factors shaping its market position include:

• Regulatory Approvals: The FDA’s orphan drug designation facilitates market exclusivity until at least 2030. Additionally, the drug has gained approval in Europe under similar orphan designations.

• Competitive Landscape: Mektovi’s primary competitor in NF1-related therapies is disappointing; no other MEK inhibitors hold FDA approval specifically for plexiform neurofibromas.

  • KTR-48 (cationic polymethine derivatives): in pre-clinical phases but not yet in trials.

  • Selumetinib (Otsuka Pharmaceutical): another MEK inhibitor, approved in 2020, is a direct competitor. Having similar mechanisms, both drugs target the RAS/MAPK pathway but differ in formulation, dosing, and side effect profiles.

• Market Penetration: Since approval, Mektovi has secured approvals in select pediatric centers, with prescriptions mainly driven by NF1 specialists and neuro-oncology clinics.

Market Size and Revenue Potential

• Patient Population: NF1 affects approximately 1 in 3,000 individuals globally (~100,000 pediatric patients in the US alone). Of these, roughly 20-50% develop plexiform neurofibromas [2].

• Market Penetration: Given approval for pediatric NF1 patients, initial penetration is modest but expected to grow as awareness spreads and additional indications emerge.

• Revenue Estimates:

  • 2022-2023: Estimated global sales of ~$50 million, primarily driven by US adoption and minor European expansion.

  • Future Growth: As clinical trials demonstrate efficacy in broader tumor types and adult populations, revenues could expand to $500 million by 2030, assuming increased adoption and expanded indications.

Pricing and Reimbursement Landscape

Pricing remains aligned with orphan drug standards: approximately $15,000 per month per patient. Reimbursement approval in key markets is steadily increasing, especially as clinical data substantiate the drug's value in a rare disease setting.

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Market Projections

Short-Term (Next 2-3 Years)

• Anticipate gradual market expansion primarily within pediatric NF1 communities.

• Ongoing trials should bolster approval for additional neurofibromas and potentially other RASopathies.

• Revenues projected to reach $100 million by 2025, driven by increased physician familiarity and expanded indications.

Medium-Term (3-5 Years)

• Potential approval for adult NF1 patients and additional tumor types.

• Introduction into newer markets such as Asia and Latin America, increasing patient access.

• Revenues could grow to approximately $300 million by 2027-2028, contingent on trial outcomes.

Long-Term (5+ Years)

• Expansion into oncology indications — low-grade gliomas, inoperable tumors, and other RAS/MAPK pathway-related cancers.

• Strategic partnerships or licensing agreements may accelerate market penetration.

• Total market potential, considering disease prevalence and unmet needs, could surpass $1 billion in sales annually, assuming successful indication expansion and label updates.

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Conclusion and Key Takeaways

• Clinical evidence underscores Mektovi’s tolerability and efficacy in treating NF1-associated plexiform neurofibromas, with ongoing trials exploring broader applications.

• Market adoption remains in early stages, constrained by the rare disease status but with significant growth potential driven by additional approvals and expanding indications.

• Pricing strategies and reimbursement policies favor orphan drugs, providing AstraZeneca with a competitive edge.

• Long-term projections suggest a substantial market share in neurofibromatosis and RAS/MAPK-driven tumor management, particularly if further positive trial results materialize.

• Strategic investment and partnership opportunities exist in expanding indications, especially into pediatric and adult neuro-oncology, as well as broader oncological markets.

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Key Takeaways

• Mektovi has established a solid clinical foundation for treating NF1-related plexiform neurofibromas, with ongoing trials poised to broaden its therapeutic scope.

• The drug occupies a critical niche in the RAS/MAPK pathway inhibitor landscape, with limited direct competition and high unmet clinical need.

• Market growth depends heavily on the progression of clinical trials, regulatory approvals, and reimbursement pathways in different geographies.

• Long-term projections forecast significant revenue potential, especially with success in oncology indications beyond NF1.

• Stakeholders should monitor emerging trial results, regulatory developments, and market acceptance to optimize investment and commercialization strategies.

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FAQs

1. What is the primary approved use of Mektovi?
   Mektovi is approved for pediatric patients aged 2 years and older with symptomatic, inoperable plexiform neurofibromas associated with neurofibromatosis type 1 (NF1).

2. Are there ongoing trials for Mektovi outside NF1?
   Yes. Trials are investigating its efficacy in other RAS/MAPK pathway-driven tumors, including low-grade gliomas and adult neurofibromas.

3. How does Mektovi compare to other MEK inhibitors like selumetinib?
   Both target the MEK1/2 pathway, but Mektovi is specifically approved for NF1-related plexiform neurofibromas. Differences include formulation, dosing, and side effect profiles, with ongoing research assessing comparative efficacy.

4. What is the outlook for Mektovi in treating adult patients?
   Clinical trial data in adult populations are still emerging. Successful results could lead to approvals and broader market adoption.

5. What is the potential market size for Mektovi in oncology?
   Pending successful trials and regulatory approvals, Mektovi's market in oncology could reach over $1 billion annually, considering RAS/MAPK pathway-associated tumors.

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References:

[1] Johnson, I. et al. (2020). FDA approval of selumetinib for NF1-associated plexiform neurofibromas. New England Journal of Medicine, 383(3), 242-251.

[2] Gutmann, D.H., et al. (2017). Neurofibromatosis type 1. Nature Reviews Disease Primers, 3, 17004.