SELUMETINIB SULFATE - Generic Drug Details

What are the generic drug sources for selumetinib sulfate and what is the scope of freedom to operate?

Selumetinib sulfate is the generic ingredient in one branded drug marketed by Astrazeneca and is included in two NDAs. There are eight patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Selumetinib sulfate has two hundred and one patent family members in forty-five countries.

One supplier is listed for this compound.

Summary for SELUMETINIB SULFATE

International Patents:	201
US Patents:	8
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	24
Clinical Trials:	37
Patent Applications:	297
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for SELUMETINIB SULFATE
DailyMed Link:	SELUMETINIB SULFATE at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for SELUMETINIB SULFATE

Constraining patent/regulatory exclusivity:

NEW PATIENT POPULATION

Dosage:

CAPSULE;ORAL

Constraining patent/regulatory exclusivity:

NEW PRODUCT

Dosage:

GRANULE;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for SELUMETINIB SULFATE

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
National Cancer Institute (NCI)	Phase 3
Sarcoma Alliance for Research through Collaboration	Phase 2
United States Department of Defense	Phase 2

See all SELUMETINIB SULFATE clinical trials

Pharmacology for SELUMETINIB SULFATE

Drug Class	Kinase Inhibitor
Mechanism of Action	Mitogen-Activated Protein Kinase Kinase 1 Inhibitors Mitogen-Activated Protein Kinase Kinase 2 Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for SELUMETINIB SULFATE

L01EE	Mitogen-activated protein kinase (MEK) inhibitors
L01E	PROTEIN KINASE INHIBITORS
L01	ANTINEOPLASTIC AGENTS
L	Antineoplastic and immunomodulating agents

US Patents and Regulatory Information for SELUMETINIB SULFATE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Astrazeneca	KOSELUGO	selumetinib sulfate	CAPSULE;ORAL	213756-002	Apr 10, 2020		RX	Yes	Yes
Astrazeneca	KOSELUGO	selumetinib sulfate	CAPSULE;ORAL	213756-002	Apr 10, 2020		RX	Yes	Yes			Y
Astrazeneca	KOSELUGO	selumetinib sulfate	CAPSULE;ORAL	213756-002	Apr 10, 2020		RX	Yes	Yes				Y

International Patents for SELUMETINIB SULFATE

Country	Patent Number	Title	Estimated Expiration
South Africa	200602588	N3 alkylated benzimidazole derivatives as MEK inhibitors
Brazil	PI0413152	derivados de benzimidazol alquilado por n3 como inibidores de mek
Poland	233177

Supplementary Protection Certificates for SELUMETINIB SULFATE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1482932	CR 2019 00011	Denmark		PRODUCT NAME: BINIMETINIB ELLER ET FARMACEUTISK ACCEPTABELT SALT ELLER SOLVAT DERAF; REG. NO/DATE: EU/1/18/1315 20180924
1482932	C01482932/01	Switzerland		PRODUCT NAME: BINIMETINIB; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 66907 01.11.2019
1482932	648	Finland

Market Dynamics and Financial Trajectory for Selumetinib Sulfate

Last updated: July 28, 2025

Introduction

Selumetinib sulfate, marketed as Koselugo, is an oral, selective MEK1/2 inhibitor developed predominantly for treating neurofibromatosis type 1 (NF1) with inoperable plexiform neurofibromas and certain other cancers. Its path from clinical trials to regulatory approval reflects evolving market conditions, clinical demand, and strategic positioning within the oncology and genetic disorder therapeutic landscapes. This report delves into the current market dynamics, financial projections, and strategic considerations shaping the future trajectory of selumetinib sulfate.

Market Overview

Selumetinib sulfate’s core indications—primarily NF1-associated plexiform neurofibromas—are rare, hereditary tumors affecting children and young adults, with an estimated prevalence of 1 in 3,000 to 4,000 individuals globally. The scarcity of effective treatments positions selumetinib within the orphan drug market, characterized by high unmet needs and premium pricing potential.

Additionally, selumetinib’s investigational pipeline extends into various solid tumors, including non-small cell lung cancer (NSCLC), melanoma, and colorectal cancers, broadening its potential market scope. Despite being approved for specific off-label uses, its market penetration remains cautiously optimistic due to regulatory and commercial factors.

Regulatory Milestones and Market Adoption

In April 2020, the U.S. Food and Drug Administration (FDA) approved selumetinib for pediatric patients aged 2 and above with symptomatic, inoperable plexiform neurofibromas [1]. This marked a significant milestone, making it one of the first targeted therapies approved for NF1-associated tumors. The designation as an orphan drug maximizes market exclusivity until 2030, providing a competitive moat.

The European Medicines Agency (EMA) approved the drug under similar indications in 2022, facilitating access across key global markets. These regulatory endorsements have catalyzed market adoption within pediatric and neuro-oncology communities. However, uptake varies by region, impacted by healthcare infrastructure, reimbursement policies, and clinician familiarity.

Market Drivers

1. Unmet Medical Needs and Clinical Benefit

NF1-associated plexiform neurofibromas can cause disfigurement and debilitating neurological symptoms. Current management relies mainly on surgical interventions, which are often insufficient or risky. Selumetinib’s demonstrated efficacy in reducing tumor volume and improving quality of life solidifies its role.

2. Orphan Drug Incentives

Market exclusivity, tax benefits, and grants associated with orphan drug designation underpin financial attractiveness. These incentives lower commercial risk and facilitate investment in manufacturing and R&D for extensions.

3. Expansion into Broader Oncology Indications

Preclinical and early clinical findings suggest selumetinib’s utility in other tumors with aberrant MEK pathway activation. Trials targeting NSCLC, melanoma, and pancreatic cancer are underway or completed, promising to enlarge its market horizon.

4. Competitive Landscape

Minimal direct competition exists for NF1-related plexiform neurofibromas, but emerging MEK inhibitors (e.g., trametinib, cobimetinib) for other indications threaten to encroach upon segments. Strategic differentiation and combination therapies will influence market share.

Market Challenges

1. Limited Patient Population

As an orphan drug targeting a rare disease, the total addressable population remains small. While this makes clinical trials feasible, it constrains revenue growth potential compared to blockbusters.

2. High Cost of Therapy

Pricing strategies reflect the drug’s orphan status and clinical efficacy. In the US, priced around $9,600 per month for pediatric patients [2], affordability and reimbursement hurdles could limit access, especially in cost-sensitive healthcare systems.

3. Long-term Data Deficiency

Long-term safety and efficacy data are limited, impacting clinician confidence and payer acceptance. Ongoing post-marketing studies aim to mitigate this barrier.

Financial Trajectory Analysis

1. Revenue Projections

Initial sales in 2021 were modest but poised for growth, fueled by expanded indications and increased awareness. Market research indicates that peak annual revenues could reach between $250 million and $500 million within five years, contingent upon market penetration, pricing strategies, and regulatory expansions [3].

2. Pricing and Reimbursement Dynamics

High per-patient charges underpin revenue streams, but payer negotiations influence price accessibility. Value-based reimbursement models may emerge, emphasizing clinical benefit and quality-of-life improvements.

3. Cost Structure and Profitability

Development, manufacturing, and marketing costs are offset by orphan drug incentives, with gross margins expected to be healthy due to limited competition. Global supply chain management will be crucial to sustain profitability.

4. Future Revenue Streams and Strategic Moves

• Pipeline expansion: Advances into additional tumors could diversify revenue sources.
• Combination therapies: Partnering with immunotherapies or chemotherapies could enhance efficacy, expanding indications.
• M&A opportunities: Larger pharma may acquire or license rights to broaden market access or accelerate clinical development.

Market Outlook and Strategic Considerations

The decade ahead hinges on clinical trial outcomes, regulatory pathways, and healthcare policy adaptations. Continued engagement with regulators and payers, coupled with targeted marketing within specialized centers, will be vital.

Innovation in delivery methods, biomarker-driven patient selection, and real-world evidence collection will influence Market Penetration and financial performance. Companies leveraging selumetinib’s niche positioning can capitalize on the growing demand for targeted, personalized therapies in oncology and genetic disorders.

Key Takeaways

• Unique Positioning: Selumetinib sulfate’s FDA and EMA approvals for NF1-related tumors secure orphan drug status, fostering exclusivity and high-margin potential despite a limited patient pool.
• Revenue Growth: Projected to reach substantial revenues in the next five years, supported by expanded indications and clinical validation.
• Market Challenges: Reimbursement negotiations, limited long-term data, and competition from emerging MEK inhibitors could temper growth.
• Strategic Opportunities: Pipeline expansion, combination therapies, and global market entry will be crucial to maximize financial trajectory.
• Regulatory and Clinical Focus: Success depends on ongoing clinical trials, real-world evidence, and proactive engagement with stakeholders.

FAQs

Q1: What is selumetinib sulfate primarily approved for?
A1: Selumetinib sulfate is approved for pediatric patients aged 2 and above with symptomatic, inoperable plexiform neurofibromas associated with neurofibromatosis type 1 (NF1).

Q2: How does orphan drug designation impact selumetinib’s market potential?
A2: Orphan drug status grants market exclusivity until 2030, tax incentives, and grants, reducing competition and allowing premium pricing, which supports revenue growth despite the small patient population.

Q3: What are the main competitive threats to selumetinib sulfate?
A3: Competitive threats include other MEK inhibitors under development or approved for different indications, as well as potential generic entrants after patent expiration, though current exclusivity limits this risk.

Q4: What are the expected financial benefits of expanding selumetinib’s indications?
A4: Broader approval in additional cancers could significantly increase revenue by tapping into larger markets, especially if clinical trials demonstrate substantial efficacy.

Q5: What strategies can optimize selumetinib’s market trajectory?
A5: Focus on clinical development, real-world evidence, strategic partnerships, targeted marketing to specialized clinicians, and health economics can enhance adoption and financial returns.

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References
[1] U.S. Food and Drug Administration. (2020). FDA approves first targeted therapy for children with a rare genetic disorder.
[2] MarketWatch. (2021). Selumetinib pricing data.
[3] GlobalData. (2022). Selumetinib market forecast and analysis report.