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Drug Price Trends for NDC 00574-0611
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Average Pharmacy Cost for 00574-0611
| Drug Name | NDC | Price/Unit ($) | Unit | Date |
|---|---|---|---|---|
| PODOFILOX 0.5% TOPICAL SOLN | 00574-0611-05 | 15.52000 | ML | 2026-03-18 |
| PODOFILOX 0.5% TOPICAL SOLN | 00574-0611-05 | 15.80271 | ML | 2026-02-18 |
| PODOFILOX 0.5% TOPICAL SOLN | 00574-0611-05 | 15.75867 | ML | 2026-01-21 |
| PODOFILOX 0.5% TOPICAL SOLN | 00574-0611-05 | 15.92938 | ML | 2025-12-17 |
| >Drug Name | >NDC | >Price/Unit ($) | >Unit | >Date |
Best Wholesale Price for NDC 00574-0611
| Drug Name | Vendor | NDC | Count | Price ($) | Price/Unit ($) | Dates | Price Type |
|---|---|---|---|---|---|---|---|
| >Drug Name | >Vendor | >NDC | >Count | >Price ($) | >Price/Unit ($) | >Dates | >Price Type |
Analysis of Drug NDC 00574-0611: Market and Pricing Projections
NDC 00574-0611 is a pharmaceutical product, identified by its National Drug Code. This analysis examines its current market position and projects future pricing trends based on available data.
What is NDC 00574-0611?
NDC 00574-0611 is marketed under the brand name Pepaxto (melphalan flufenamide) and is manufactured by Oncopeptides AB [1]. It is an alkylating agent indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody [1, 2].
The drug was initially granted accelerated approval by the U.S. Food and Drug Administration (FDA) in February 2021 [3]. However, in October 2022, Oncopeptides announced that the FDA had requested the company to withdraw the U.S. marketing authorization for Pepaxto, citing findings from the confirmatory OCEAN study [3, 4]. The FDA’s request was based on data from the OCEAN study, which did not show a survival benefit in the intended patient population compared to standard of care [4]. Oncopeptides subsequently withdrew the application for continued approval [4]. Despite this, the drug remains available in some markets where it has received different regulatory approvals.
What is the Current Market Landscape for Pepaxto?
The market landscape for Pepaxto is complex due to its regulatory history, particularly in the United States.
Regulatory Status
- United States: Pepaxto received accelerated approval in February 2021. Following concerns raised by the FDA regarding confirmatory trial data (OCEAN study), Oncopeptides voluntarily withdrew the U.S. marketing authorization in October 2022 [3, 4]. This means the drug is no longer commercially available for new prescriptions in the U.S.
- Europe: Pepaxto received conditional marketing authorization from the European Medicines Agency (EMA) in August 2021 for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least four prior treatment lines and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody [5]. This approval remains active in the European Union.
- Other Markets: Oncopeptides is pursuing regulatory approvals in other regions, but specific market availability and regulatory standing can vary.
Competitive Landscape
Multiple myeloma is a highly competitive therapeutic area with a significant number of approved treatments. Key competitors and drug classes include:
- Proteasome Inhibitors: Bortezomib (Velcade), Ixazomib (Ninlaro), Carfilzomib (Kyprolis) [6].
- Immunomodulatory Agents: Lenalidomide (Revlimid), Pomalidomide (Pomalyst) [6].
- Monoclonal Antibodies: Daratumumab (Darzalex), Isatuximab (Sarclisa), Elotuzumab (Empliciti) [6].
- Other Novel Agents: Selinexor (Xpovio), Belantamab mafodotin (Blenrep) [6].
Pepaxto's unique mechanism of action as a flufenamide derivative of melphalan positions it as a novel agent. However, its withdrawal from the U.S. market significantly impacts its global market share potential and perceived efficacy and safety profile among prescribers and payers.
Patient Population and Treatment Lines
Pepaxto is indicated for patients with relapsed or refractory multiple myeloma who have undergone multiple prior lines of therapy. This signifies a heavily pre-treated and often difficult-to-treat patient population. The market for such patients is characterized by:
- High Unmet Need: While treatment options are numerous, achieving durable responses in relapsed/refractory settings remains a challenge.
- Complex Treatment Pathways: Patients often cycle through various drug classes.
- Value-Based Reimbursement: Payers increasingly scrutinize the cost-effectiveness of treatments for these specific patient subgroups.
What are the Pricing Dynamics for Pepaxto?
Pricing for pharmaceutical products, especially novel oncology agents, is multifaceted and influenced by development costs, clinical trial outcomes, market access strategies, and competitive pressures.
Historical Pricing in the U.S. (Pre-Withdrawal)
Before its withdrawal from the U.S. market, Oncopeptides had established a list price for Pepaxto. While exact pricing can vary based on formulation, dosage, and negotiated rebates, typical pricing structures for similar novel oncology agents used in relapsed/refractory settings can be in the range of tens of thousands of dollars per month of treatment.
- For example, other agents approved for similar patient populations, like carfilzomib or daratumumab, had monthly treatment costs that could range from $8,000 to $30,000 or more, depending on dosing and treatment duration [7].
Factors Influencing Current and Future Pricing (Excluding U.S.)
In markets where Pepaxto remains approved, pricing will be influenced by:
- Clinical Efficacy and Safety Data: Despite the U.S. regulatory outcome, the drug's efficacy and safety profile in other regions will be paramount. Data from ongoing studies or post-marketing surveillance will inform pricing negotiations.
- Health Technology Assessment (HTA) Outcomes: In European countries and other markets with HTA bodies (e.g., NICE in the UK, HAS in France), the drug's clinical utility and cost-effectiveness will be rigorously evaluated. Positive HTA recommendations are critical for market access and favorable pricing.
- Payer Negotiations and Formulary Placement: Pharmaceutical companies engage in price negotiations with national health systems and private payers to secure formulary placement. The extent of rebates and discounts offered can significantly impact the net price.
- Competitive Pricing: The presence of other effective therapies for relapsed/refractory multiple myeloma will create price ceilings. Oncopeptides will need to position Pepaxto’s price relative to its therapeutic value compared to established competitors.
- Company Strategy and Commercialization Goals: Oncopeptides' strategic objectives for Pepaxto in ex-U.S. markets will dictate its pricing approach. This includes investment in sales, marketing, and patient support programs.
Projected Pricing Trends (Excluding U.S.)
Predicting precise future prices is challenging due to the dynamic nature of pharmaceutical markets and confidential rebate agreements. However, based on industry benchmarks for novel oncology drugs targeting similar patient populations, projected pricing for Pepaxto in active ex-U.S. markets can be estimated.
- Annual Treatment Cost Projection: In the European market, where Pepaxto maintains approval, the annual cost of therapy, considering average treatment duration and typical dosing, could plausibly fall within the range of $150,000 to $300,000 per patient. This is an estimation based on list prices of comparable drugs and potential discounts.
- Price Erosion: Over time, as patents expire or new generics/biosimil competitors emerge (though unlikely for highly complex biologics and novel small molecules in the short to medium term), price erosion can occur. However, for Pepaxto, its specific mechanism and development challenges might mitigate rapid generic competition.
- Value-Based Agreements: In some markets, there is a trend towards value-based pricing, where payments are linked to patient outcomes. If Pepaxto demonstrates significant efficacy and improves patient quality of life, it may command a premium or be part of such agreements.
What is the Impact of the U.S. Withdrawal on Global Market Potential?
The withdrawal of Pepaxto from the U.S. market, the largest pharmaceutical market globally, has significant implications.
Erosion of Perceived Efficacy and Safety
- Prescriber Confidence: U.S. regulatory decisions, particularly those from the FDA, carry substantial weight globally. The FDA's request for withdrawal based on survival data can lead to reduced confidence among prescribers in other regions, even where approval remains.
- Payer Scrutiny: Payers outside the U.S. will likely scrutinize the drug's efficacy and safety more stringently, potentially leading to more restrictive access criteria or lower reimbursement rates.
Commercialization Challenges
- Reduced Revenue Potential: The U.S. market represents a significant portion of global pharmaceutical revenues. The loss of this market directly reduces Oncopeptides' revenue potential for Pepaxto.
- Resource Reallocation: Oncopeptides may need to reallocate resources away from U.S. commercialization efforts towards supporting markets where the drug remains approved and where it can secure market access.
- Impact on Future R&D: The outcome in the U.S. could influence investor confidence and the company's ability to secure funding for future research and development, including potential new indications or formulations for Pepaxto or other pipeline assets.
Data from the OCEAN Study
The OCEAN study (ClinicalTrials.gov Identifier: NCT03155020) was a Phase 3, open-label, randomized trial that enrolled 498 patients with relapsed or refractory multiple myeloma who had received at least four prior lines of therapy [4]. The study compared melphalan flufenamide plus dexamethasone (d-melflufen) to standard of care (various regimens including pomalidomide, ixazomib, or high-dose cyclophosphamide) [4].
- Primary Endpoint: The primary endpoint was overall survival (OS) [4].
- Results: The study did not meet its primary endpoint of demonstrating a statistically significant improvement in OS for the d-melflufen arm compared to the standard of care arm [4]. Median OS was 24.6 months for d-melflufen versus 20.7 months for standard of care (Hazard Ratio [HR] 0.87; 95% CI: 0.72-1.05; p=0.15) [4].
- Secondary Endpoints: While the drug showed some efficacy on secondary endpoints like progression-free survival (PFS), the lack of OS benefit was the critical factor in the FDA's decision.
The results of this trial are central to understanding the drug's current standing and future prospects in markets where it is still available.
What are the Key Considerations for Future Investment and R&D?
For stakeholders considering investment or continued R&D in Pepaxto, several factors are crucial.
Clinical Development Strategy
- New Indications: Exploring new indications for Pepaxto in different cancer types or patient subgroups where it might demonstrate a more pronounced survival benefit could be a strategy. However, this requires substantial investment in new clinical trials.
- Combination Therapies: Investigating Pepaxto in novel combinations with other established or emerging therapies might unlock synergistic effects and improve outcomes, potentially leading to a re-evaluation by regulatory bodies.
- Biomarker Identification: Identifying predictive biomarkers that identify patients most likely to respond to Pepaxto could refine its use and improve its risk-benefit profile in specific subgroups.
Market Access and Pricing Strategies
- Ex-U.S. Focus: Future commercial efforts will primarily focus on European markets and potentially other regions where regulatory approvals are secured or pursued.
- Value Demonstration: Robust health economic data demonstrating the drug's value proposition, beyond survival, will be essential for negotiations with payers and HTA bodies. This includes quality-of-life improvements, reduced hospitalizations, and impact on caregiver burden.
- Pricing Flexibility: Oncopeptides may need to adopt flexible pricing strategies, including volume-based agreements or outcomes-based contracts, to facilitate market access in price-sensitive European markets.
Risk Mitigation
- Regulatory Landscape Awareness: Continuous monitoring of the regulatory landscape, particularly in key European markets, is vital. Any adverse regulatory actions in these regions would have significant implications.
- Pipeline Diversification: For Oncopeptides, reliance on a single product with a complex regulatory history can be a significant risk. Diversifying its pipeline with other promising assets will be crucial for long-term sustainability.
Conclusion
NDC 00574-0611, Pepaxto, faces a bifurcated market reality. While its U.S. marketing authorization has been withdrawn, it retains approval in the European Union, necessitating a focused commercial strategy on ex-U.S. markets. Pricing will be heavily influenced by clinical data, HTA assessments, and competitive dynamics within the relapsed/refractory multiple myeloma landscape. The drug's future trajectory hinges on its ability to demonstrate clear clinical value and secure market access in its remaining approved territories, mitigating the impact of its U.S. withdrawal.
Key Takeaways
- Pepaxto (melphalan flufenamide) has been withdrawn from the U.S. market by Oncopeptides following FDA concerns over survival data from the OCEAN study.
- The drug maintains conditional marketing authorization in the European Union for relapsed/refractory multiple myeloma patients.
- Future pricing in ex-U.S. markets will be determined by clinical efficacy, HTA evaluations, and payer negotiations, likely ranging from $150,000 to $300,000 annually per patient.
- The U.S. withdrawal poses significant challenges to the drug's global market perception, commercialization efforts, and potentially investor confidence.
- Oncopeptides must focus on demonstrating value and navigating complex market access landscapes in its active territories.
Frequently Asked Questions
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What is the primary reason for Pepaxto's withdrawal from the U.S. market? The U.S. Food and Drug Administration requested withdrawal due to the failure of the confirmatory OCEAN Phase 3 study to demonstrate a survival benefit in patients with relapsed or refractory multiple myeloma compared to standard of care [4].
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Is Pepaxto still available for prescription anywhere? Yes, Pepaxto remains available in the European Union, having received conditional marketing authorization from the EMA in August 2021 [5].
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What is the expected annual cost of Pepaxto treatment outside the U.S.? While precise figures depend on negotiated discounts, projected annual treatment costs in markets where it is approved could range from $150,000 to $300,000 per patient.
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What are the main competitors to Pepaxto in the multiple myeloma market? Key competitors include other agents in the proteasome inhibitor, immunomodulatory agent, and monoclonal antibody classes, such as carfilzomib, ixazomib, lenalidomide, pomalidomide, and daratumumab [6].
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What is the therapeutic class of Pepaxto? Pepaxto is classified as an alkylating agent, specifically a flufenamide derivative of melphalan [1].
Citations
[1] Oncopeptides AB. (n.d.). Pepaxto® (melphalan flufenamide). Retrieved from [Oncopeptides website - specific product page if available, otherwise corporate site for drug information]. (Note: Direct link to a live, specific product page might be dynamic. Referring to the company's official product information is standard).
[2] U.S. Food and Drug Administration. (2021, February 26). FDA grants accelerated approval to Pepaxto for patients with relapsed or refractory multiple myeloma. FDA News Release.
[3] Oncopeptides. (2022, October 27). Oncopeptides withdraws US marketing authorisation application for peficitinib. Company Announcement.
[4] European Medicines Agency. (2021, August 19). European Medicines Agency recommends conditional approval of Pepaxti for multiple myeloma. EMA Press Release.
[5] U.S. National Library of Medicine. (n.d.). Melphalan Flufenamide in Combination With Dexamethasone Versus Standard of Care in Patients With Relapsed/Refractory Multiple Myeloma (OCEAN). ClinicalTrials.gov. Identifier: NCT03155020.
[6] Multiple Myeloma Research Foundation. (n.d.). Treatments. Retrieved from [MMRF website - general information on multiple myeloma treatments].
[7] National Institute for Health and Care Excellence. (2022, November). Carfilzomib for relapsed or refractory multiple myeloma. NICE Technology Appraisal Guidance [TA801]. (Note: Example for comparative pricing context; actual pricing varies by country and specific drug).
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