Last updated: February 13, 2026
What is NDC 00046-0872?
NDC 00046-0872 corresponds to Selumetinib, marketed under the brand name Koselugo. It is an oral MEK inhibitor approved by the FDA in April 2020 for treating pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1)–related plexiform neurofibromas.
Market Landscape
Market Size and Demand
- Patient population: NF1 affects approximately 1 in 3,000 individuals globally; about 20-30% develop plexiform neurofibromas.
- Target population: Children aged 2 years and older diagnosed with NF1-related plexiform neurofibromas. Estimated annual treatable cases in the U.S.: approximately 10,000–15,000 patients.
Competition
- No approved targeted therapies directly treat plexiform neurofibromas.
- Off-label treatments: Chemotherapy, radiation, or surgical approaches.
- Potential pipeline drugs:
- MEK inhibitors: Other candidates like Trametinib (GSK/Novartis) under investigation.
- Emerging therapies: Novel agents targeting NF1 pathways.
Market Drivers
- FDA approval in 2020 increased clinical adoption.
- Physician awareness of Selumetinib’s targeted efficacy.
- Reimbursement frameworks: CMS and private insurers began coverage in 2021.
Barriers
- High treatment cost: The annual cost per patient exceeds $100,000.
- Limited long-term data: Concerns about safety and durability.
- Pricing negotiations: Negotiations with payers could influence market penetration.
Pricing History and Projections
Current Pricing
- List price: Approximately $115,000 to $125,000 annually per patient (per drug labels and publicly available pricing in 2023).
- Pricing structure: Based on weight-based dosing, common in pediatric oncology drugs.
- Reimbursement: Secured through commercial insurers, Medicaid, and Medicare under paid claims.
Price Trends
- Initial launch (2020): Launch price of around $116,000 annually.
- Price adjustments: Insignificant in first two years; prices remained stable.
Projections
| Year |
Estimated Price Range |
Notes |
| 2023 |
$115,000 - $125,000 |
Stable, with potential for minor adjustments |
| 2024 |
$115,000 - $125,000 |
Maintains stable pricing; payor negotiations could influence |
| 2025 |
$113,000 - $123,000 |
Possible discounts or rebates as market matures |
| 2026 |
$113,000 - $120,000 |
Competition from pipeline and biosimilars could exert pressure |
Long-term prices may decline due to increased competition, biosimilars, or changes in reimbursement policies. The absence of direct competitors keeps prices relatively stable through at least 2025.
Financial and Market Impact Analysis
- Revenue potential: With approximately 12,500 treated patients in the U.S. at average $120,000 per patient, the US market size is roughly $1.5 billion annually.
- Global markets: Europe, Japan, and other countries contribute an additional 30-40% in revenue, assuming similar pricing and uptake.
- Market penetration: Estimated 80-85% of eligible patients by 2025, driven by increased recognition and insurance coverage.
Regulatory and Policy Considerations
- Pricing regulations: Tied to healthcare reforms, with increasing pressure for value-based pricing.
- Orphan drug status: Provides market exclusivity until 2027 in the U.S., helping maintain pricing power.
- Pricing thresholds: May face scrutiny from government agencies aiming to control drug costs.
Key Takeaways
- NDC 00046-0872 (Selumetinib) has a niche but expanding market due to lack of alternatives for NF1-related plexiform neurofibromas.
- Current list prices are stable around $115,000–$125,000 annually, with expectations of minor declines from 2025 onward.
- Revenue prospects remain high, with US estimates exceeding $1.2 billion annually under current conditions.
- Competitive pressure remains limited for now but could intensify with pipeline developments.
- Reimbursement and pricing policies will significantly influence long-term market access and pricing.
FAQs
1. What is the primary indication for NDC 00046-0872?
Treatment of pediatric patients aged 2 and older with neurofibromatosis type 1–related plexiform neurofibromas.
2. How does the price compare to similar pediatric therapies?
Selumetinib’s annual cost (~$120,000) aligns with other targeted oncology drugs for rare pediatric conditions, which typically cost between $100,000 to $150,000.
3. What factors could impact future price projections?
Introduction of biosimilars or alternative therapies, changes in insurance reimbursement policies, and regulatory adjustments.
4. What is the market penetration potential?
Approximately 80-85% of eligible patients are expected to receive treatment by 2025, assuming stable approval and reimbursement.
5. How does regulatory exclusivity impact pricing?
Market exclusivity until 2027 allows for pricing stability and limited competition during that period.
References
- U.S. Food and Drug Administration. FDA approves Koselugo for neurofibromatosis type 1. 2020.
- IQVIA. Healthcare Market Estimates. 2023.
- CMS.gov. Coverage policies for orphan drugs. 2021.
- Drug pricing databases and hospital formularies, 2023.
[1] FDA Approval Announcement: https://www.fda.gov/news-events/press-announcements/fda-approves-koselugo-orphan-drug-treatment-children-neurofibromatosis-1-continue-its-expansion
