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Last Updated: January 30, 2026

Drug Price Trends for NDC 00409-6625


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Best Wholesale Price for NDC 00409-6625

These are wholesale prices available to the US Federal Government which, by law, must be the best prices available under comparable terms and conditions
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Market Analysis and Price Projections for NDC 00409-6625

Last updated: August 4, 2025


Introduction

The drug identified by NDC 00409-6625 corresponds to Kynamro (mipomersen sodium), a medication developed by Ionis Pharmaceuticals and approved by the U.S. Food and Drug Administration (FDA) for the treatment of homozygous familial hypercholesterolemia (HoFH). This analysis provides an in-depth review of the current market landscape, competitive positioning, pricing trends, and future projection strategies relevant to Kynamro.


Regulatory and Therapeutic Context

Kynamro (mipomersen) is an antisense oligonucleotide designed to reduce low-density lipoprotein (LDL) cholesterol levels in patients with HoFH unresponsive to existing therapies. Approved in 2013, its market exclusivity pertains primarily to its indication for a rare disease, inherently limiting the number of eligible patients.

The drug's clinical indication targets a small patient population, estimated around 1,000-1,500 individuals in the U.S., representing a niche but high-need market.[1] Its mechanism, targeting apolipoprotein B (ApoB) synthesis, distinguishes it from traditional lipid-lowering agents, positioning it uniquely albeit with significant administration and safety considerations.


Market Overview

1. Market Size & Epidemiology

The HoFH patient population remains small but significant due to the severity of disease and limited treatment options. According to the American Heart Association, HoFH incidence is approximately 1 in 1 million globally, with higher prevalence in certain populations due to founder effects.[2]

2. Competitive Landscape

Kynamro faces competition from other lipid-lowering therapies, notably PCSK9 inhibitors (evolocumab, alirocumab, etc.), which, though approved for heterozygous familial hypercholesterolemia and general hyperlipidemia, are increasingly used off-label for Homozygous cases. Additionally, emerging novel therapies, such as angiopoietin-like protein 3 (ANGPTL3) inhibitors, promise future competition.

Given the targeted therapeutic approach, few direct competitors exist in the HoFH indication. However, the efficacy, safety profile, and route of administration significantly influence market penetration.

3. Market Penetration Factors

Factors influencing Kynamro's market penetration include:

  • Efficacy and safety concerns: Kynamro's boxed warning for hepatotoxicity and injection site reactions limits its widespread adoption.
  • Administration route: Weekly subcutaneous injections pose adherence challenges, especially compared to oral therapies.
  • Pricing and reimbursement: Price sensitivity and payor coverage significantly impact patient access in a high-cost, narrow-use setting.

Current Pricing Landscape

1. Wholesale Acquisition Cost (WAC) and List Price

At approval, Kynamro's list price was approximately $450,000 to $500,000 per year for a typical patient.[3] This high cost reflects its specialized indication, complex manufacturing, and limited patient population. Actual net prices often vary due to discounts, rebates, and payor negotiations.

2. Cost-Effectiveness and Reimbursement

Cost-utility analyses demonstrate challenges in profitability given the small population and high treatment costs. Insurance providers often scrutinize reimbursement applications, which can impact available patient access and overall market size.

3. Price Trends and Access

Limited data suggest the price remains stable, with minor adjustments driven by inflation, market negotiations, and formulary positioning. The high price point constrains broad utilization but sustains profitability within its niche.


Forecasted Market Trends and Price Projections

1. Near-Term Outlook (Next 1-3 Years)

  • Stable pricing: Given the lack of significant competition specifically targeting HoFH, the price is expected to remain relatively stable barring policy shifts or new competitors.
  • Market growth: Modest growth anticipated, driven primarily by increased diagnosis and provider familiarity rather than price escalation.
  • Reimbursement dynamics: Payers may negotiate better rebates or impose utilization management to control costs, influencing net pricing.

2. Mid to Long-Term Projections (3-7 Years)

  • Introduction of Competitors: The pipeline for antisense therapies, gene editing (e.g., CRISPR-Cas9-based treatments), and novel lipid-lowering agents could challenge Kynamro's market exclusivity.
  • Pricing pressure: As newer therapies demonstrate improved safety and convenience, Kynamro's high price may lead to downward pressure.
  • Label Expansion: Potential approval for broader lipid disorders could expand patient eligibility, possibly impacting price and market volume.

3. Impact of Biosimilars and Follow-on Products

While biosimilars are unlikely in the short term due to the nature of antisense oligonucleotides, evolving biosimilar policies and technological advancements may influence the landscape in the future, possibly reducing costs.


Legal and Policy Landscape Impacting Pricing

Recent trends indicate heightened scrutiny of high-cost specialty drugs. Legislation targeting drug pricing transparency, value-based pricing models, and increased use of risk-sharing agreements can influence the market dynamics of Kynamro.

Furthermore, orphan drug incentives provide market exclusivity periods, generally 7 years in the U.S., supporting sustained pricing. However, potential legislative shifts aimed at curbing outlier drug prices could challenge future pricing stability.


Market Opportunities and Risks

Opportunities:

  • Market niche dominance due to the unmet need.
  • Potential label expansion, increasing eligible patient population.
  • Partnerships with specialty pharmacies to enhance access.

Risks:

  • Emergence of novel gene therapies offering durable or curative solutions.
  • Reimbursement constraints driven by high costs.
  • Adverse safety profiles or dosing complexities.

Concluding Summary

Kynamro maintains a high-price, niche market position primarily due to its specialized indication and limited competition. Its valuation hinges on continued clinical efficacy, safety, and payer acceptance. While short-term outlooks predict stable pricing aligned with current standards, longer-term projections suggest potential downward pricing pressures as innovation accelerates in lipid management.


Key Takeaways

  • The small but high-need population for homozygous familial hypercholesterolemia sustains Kynamro’s premium pricing.
  • Clinical safety concerns and administration mode impede broader adoption, influencing overall revenue.
  • Competitive advances, particularly gene editing therapies, threaten the future market share and pricing stability.
  • Reimbursement negotiations and healthcare policies will significantly impact market access and net pricing.
  • Expect stable pricing in the near term, with potential declines driven by technological innovation and policy reforms over the next 5–7 years.

FAQs

1. How does the rarity of HoFH impact Kynamro’s pricing strategy?
The ultra-rare disease status grants market exclusivity and supports high pricing, as the small patient population justifies significant R&D investments and regulatory incentives.

2. Are there any upcoming competitors that could disrupt Kynamro’s market?
Emerging gene therapies and next-generation antisense oligonucleotides are under development, potentially offering more durable or safer treatment options and reducing reliance on Kynamro.

3. How do reimbursement policies influence Kynamro’s market penetration?
High treatment costs and safety concerns lead to variable reimbursement, with payors potentially limiting access through prior authorization and formulary restrictions, impacting revenue.

4. What factors could lead to a significant reduction in Kynamro’s price?
Introduction of more effective or safer alternatives, policy reforms targeting high-cost drugs, and negotiations resulting in favorable rebates could all drive prices down.

5. How might label expansion affect Kynamro’s market and pricing?
Expanding approval to broader hyperlipidemia populations could increase market size, potentially allowing for price adjustments—either increased, due to higher volume, or decreased, due to competitive pressures.


References

[1] FDA. (2013). Kynamro (mipomersen) approval letter.
[2] American Heart Association. (2014). Epidemiology of familial hypercholesterolemia.
[3] Bloomberg Intelligence. (2022). High-cost therapies overview.

Note: All projections and analyses are subject to change based on future regulatory developments, scientific breakthroughs, and market dynamics.

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