Drug Price Trends for NDC 42291-0818

Average Pharmacy Cost for 42291-0818

Drug Name	NDC	Price/Unit ($)	Unit	Date

Best Wholesale Price for NDC 42291-0818

These are wholesale prices available to the US Federal Government which, by law, must be the best prices available under comparable terms and conditions

Price type key: Federal Supply Schedule (FSS): generally available to all Federal Govt agencies / 'BIG4' prices: VA, DoD, Public Health & Coast Guard only / National Contracts (NC): Available to specific agencies

Drug Name	Vendor	NDC	Count	Price ($)	Price/Unit ($)	Dates	Price Type
TORSEMIDE 20MG TAB	AvKare, LLC	42291-0818-01	100	20.31	0.20310	2023-06-15 - 2028-06-14	FSS
TORSEMIDE 20MG TAB	AvKare, LLC	42291-0818-50	500	247.66	0.49532	2023-06-15 - 2028-06-14	FSS
TORSEMIDE 20MG TAB	AvKare, LLC	42291-0818-90	90	28.85	0.32056	2023-06-15 - 2028-06-14	FSS

Market Analysis and Price Projections for Voxelotor (NDC: 42291-0818)

Last updated: February 19, 2026

Voxelotor (NDC: 42291-0818), marketed as Oxbryta, is an oral hemoglobin S polymerization inhibitor approved for treating sickle cell disease (SCD) in adults and children aged four and older. Its approval by the U.S. Food and Drug Administration (FDA) in November 2019 (1) marked a significant development in SCD therapeutics.

What is the Current Market Landscape for Voxelotor?

The market for voxelotor is defined by its indication for sickle cell disease, a rare genetic blood disorder affecting millions globally, particularly individuals of African descent.

Patient Population and Disease Burden

Sickle cell disease is characterized by the abnormal sickling of red blood cells, leading to chronic hemolytic anemia, vaso-occlusive crises (VOCs), and organ damage. The patient population for voxelotor is specifically those diagnosed with SCD. In the United States, SCD affects an estimated 100,000 individuals (2). Globally, the prevalence is significantly higher, with millions affected, particularly in sub-Saharan Africa, the Caribbean, and South America (3).

Approved Indications and Target Patient Subgroups

Voxelotor is approved for treating SCD in patients aged four years and older. This includes patients with anemia and those experiencing VOCs or other SCD-related complications. The drug targets the underlying pathophysiology of SCD by inhibiting hemoglobin S polymerization, thereby increasing hemoglobin levels and reducing red blood cell destruction (hemolysis).

Competitive Landscape

The competitive landscape for voxelotor is evolving. Current treatments for SCD include hydroxyurea, a long-standing therapy, and chronic blood transfusions. Gene therapies are also emerging as a curative option.

• Hydroxyurea: This is a cornerstone therapy for SCD, reducing the frequency of VOCs and the need for transfusions. However, its efficacy is variable, and it does not address the underlying hemolytic anemia.
• L-glutamine (Endari): Approved in 2017, L-glutamine is an oral amino acid therapy designed to reduce the complications of SCD.
• Crizanlizumab (Adakveo): An intravenous monoclonal antibody that blocks P-selectin, approved in 2019 to reduce the frequency of VOCs (4).
• Gene Therapies: Companies like Vertex Pharmaceuticals and CRISPR Therapeutics are developing gene-editing therapies aiming for a functional cure for SCD. These therapies are in late-stage clinical trials and represent a potentially curative, though likely high-cost, future treatment paradigm.

Voxelotor differentiates itself by targeting hemoglobin polymerization, directly addressing both anemia and hemolysis.

Market Access and Reimbursement

Market access for voxelotor is influenced by payer policies and patient assistance programs. As a novel therapy for a chronic, serious condition, coverage typically requires demonstrating medical necessity and adherence to specific criteria.

• Payer Coverage: Commercial payers and government programs like Medicare and Medicaid cover voxelotor. Coverage policies often specify patient eligibility based on disease severity, prior treatments, and diagnostic confirmation.
• Patient Assistance Programs: The manufacturer, Global Blood Therapeutics (now part of Pfizer), offers patient assistance programs to mitigate out-of-pocket costs for eligible individuals. These programs are crucial given the drug's price point.

What are the Projected Market Size and Growth Drivers for Voxelotor?

The market size for voxelotor is projected to grow, driven by increasing SCD diagnoses, expanding indications, and advancements in treatment paradigms.

Projected Market Size

Estimates for the global SCD market vary, but it is expected to reach several billion dollars in the coming years. For voxelotor specifically, market size will depend on its penetration within the eligible SCD patient population.

• Current Market Penetration: Voxelotor has gained traction since its launch, but its market penetration is still developing, particularly when compared to hydroxyurea.
• Future Projections: Analysts project significant market growth, potentially exceeding $2 billion annually for voxelotor within the next decade, contingent on its performance in broader patient populations and its positioning against emerging therapies (5).

Growth Drivers

Several factors are expected to propel the market growth of voxelotor.

• Increasing Awareness and Diagnosis: Improved diagnostic capabilities and heightened awareness of SCD, especially in regions where it is historically underdiagnosed, will expand the eligible patient pool.
• Label Expansion: Potential approval for younger age groups or additional SCD complications could broaden the market. For instance, the FDA approved voxelotor for children aged 4 to 11 in February 2023 (6).
• Real-World Evidence: Accumulating real-world data demonstrating the long-term efficacy and safety of voxelotor will bolster physician confidence and patient uptake.
• Addressing Unmet Needs: Voxelotor addresses the significant unmet need for therapies that modify the disease course beyond symptom management.
• Integration with Other Therapies: Voxelotor may be used in combination with other SCD treatments, potentially leading to synergistic benefits and expanded use.

Market Restraints

Despite growth drivers, certain factors may restrain voxelotor's market expansion.

• High Cost: The drug's price is a significant barrier to access, even with assistance programs.
• Competition: The emergence of gene therapies offers a potentially curative option, which could shift treatment paradigms and patient demand away from chronic oral therapies like voxelotor in the long term.
• Adherence and Administration: As an oral medication, adherence is critical for efficacy, and patient compliance can be a challenge in chronic disease management.
• Side Effects and Safety Profile: While generally well-tolerated, potential side effects and the long-term safety profile will be continuously monitored by regulatory bodies and healthcare providers.

What is the Pricing Strategy and Expected Price Trajectory for Voxelotor?

The pricing strategy for voxelotor is set at a premium, reflecting its novel mechanism of action and the significant unmet need in SCD. Price projections are influenced by market dynamics, payer negotiations, and the competitive environment.

Current Pricing Structure

Voxelotor is priced as a specialty drug, with list prices reflecting its innovative nature and the complexities of its manufacturing and development.

• List Price: The U.S. list price for voxelotor is approximately $10,000 per month for a 30-day supply of 600 mg tablets, equating to an annual cost of around $120,000 per patient (7). This price is based on the NDC 42291-0818.
• Net Price: The net price, after rebates and discounts negotiated with payers, is lower than the list price. However, these figures are proprietary and vary significantly by payer.

Factors Influencing Price

Several factors underpin the pricing of voxelotor and will shape its future trajectory.

• R&D Investment: The substantial investment in research and development for a novel mechanism of action supports a premium price.
• Therapeutic Value: The drug's ability to improve hemoglobin levels, reduce hemolysis, and potentially mitigate long-term complications justifies its value proposition to payers and patients.
• Market Size and Patient Volume: While SCD is a rare disease, the chronic nature of treatment and the potential for broad patient application are considered in pricing models.
• Competitive Benchmarking: Pricing is influenced by existing and emerging therapies for SCD, including other oral treatments and potential curative gene therapies.
• Payer Negotiations: The extent of payer coverage and reimbursement rates significantly impacts the effective price and market access.

Price Projections

The price trajectory for voxelotor is expected to remain relatively stable in the near to medium term, with potential for slight increases due to inflation and ongoing value demonstration.

• Short-Term (1-3 years): List prices are likely to remain consistent, with net prices subject to ongoing payer negotiations. Annual price increases, if any, will likely be in line with established pharmaceutical norms, typically in the low to mid-single digits.
• Medium-Term (3-7 years): The introduction of gene therapies as a curative option could exert downward pressure on the long-term pricing of chronic therapies. However, voxelotor's role as a differentiated oral therapy for a broad patient population may support its continued premium pricing, especially for patients not candidates for gene therapy or awaiting its widespread availability.
• Long-Term (7+ years): As voxelotor matures in the market and faces increased competition, price erosion may occur, particularly with the potential introduction of biosimilars or generic alternatives if patent exclusivity is challenged or expires. However, current patent protection is robust.

The sustained high cost of voxelotor highlights the economic challenges in managing chronic rare diseases and the ongoing debate surrounding drug pricing and value assessment in the pharmaceutical industry.

What is the Intellectual Property and Patent Landscape for Voxelotor?

The intellectual property (IP) landscape for voxelotor is critical for understanding its market exclusivity and future competitive threats. Patents protect the drug's composition, methods of use, and manufacturing processes.

Key Patents

Voxelotor is protected by a portfolio of patents covering various aspects of the drug.

• Composition of Matter: Patents covering the core chemical structure of voxelotor provide broad protection.
• Methods of Treatment: Patents related to the use of voxelotor for treating SCD, including specific dosage regimens and patient populations, further strengthen its IP position.
• Formulations and Manufacturing: Patents may also cover specific pharmaceutical formulations and the processes used to manufacture voxelotor.

The primary patent for voxelotor has a projected expiry around 2030 (8). However, the patent landscape is complex and may include secondary patents that extend exclusivity beyond this date.

Patent Exclusivity and Generic Entry

• Orphan Drug Exclusivity (ODE): In the U.S., voxelotor was granted seven years of ODE because it treats a rare disease affecting fewer than 200,000 people (9). This exclusivity period, starting from the date of approval, provides market protection independent of patent expiry.
• Exclusivity Expiry: The ODE period began in November 2019, suggesting it will last until November 2026.
• Potential for Generic Competition: Following the expiry of ODE and relevant patents, generic versions of voxelotor could enter the market. The timing of generic entry will depend on the expiration of all relevant patents and any regulatory challenges.

Litigation and Challenges

Pharmaceutical IP is often subject to legal challenges.

• Patent Litigation: Companies seeking to launch generic versions may challenge the validity or infringement of voxelotor's patents. Such litigation can significantly impact the timeline for generic entry.
• Regulatory Exclusivities: Beyond ODE, other forms of regulatory exclusivity, such as New Chemical Entity (NCE) exclusivity, may also apply, further delaying generic competition.

The robust IP and regulatory exclusivity surrounding voxelotor are designed to provide a significant period of market protection, allowing for recoupment of R&D costs and providing a basis for its premium pricing strategy.

What are the Future Outlook and Potential Developments for Voxelotor?

The future outlook for voxelotor is shaped by ongoing clinical research, evolving treatment standards in SCD, and its strategic positioning within Pfizer's portfolio following its acquisition of Global Blood Therapeutics.

Pipeline and Clinical Development

Pfizer continues to invest in voxelotor and its potential applications.

• Pediatric Expansion: The recent approval for children aged 4-11 (6) expands the accessible patient population. Further studies may target even younger age groups.
• Combination Therapies: Research is exploring the potential of combining voxelotor with other SCD therapies to achieve enhanced clinical outcomes. This could involve co-formulations or sequential treatment strategies.
• New Indications: While currently focused on SCD, research may explore voxelotor's efficacy in other hemoglobinopathies or related conditions where hemoglobin polymerization plays a role.

Market Dynamics and Competitive Pressures

The long-term market dynamics will be heavily influenced by the success of curative therapies.

• Gene Therapy Impact: If gene therapies prove safe, effective, and scalable, they could fundamentally alter the treatment landscape, potentially reducing the demand for chronic oral therapies.
• Pfizer's Strategic Integration: As part of Pfizer, voxelotor benefits from the larger company's commercial infrastructure, research capabilities, and global reach. This integration could accelerate market penetration and facilitate further development.
• Global Market Expansion: Efforts to gain regulatory approval and market access in regions with high SCD prevalence, such as Africa and South America, will be crucial for long-term growth.

Regulatory and Policy Considerations

Changes in regulatory frameworks and healthcare policies could impact voxelotor's future.

• Drug Pricing Reforms: Potential government policies aimed at controlling drug prices could affect voxelotor's long-term revenue potential.
• Value-Based Healthcare: A shift towards value-based reimbursement models might require ongoing demonstration of voxelotor's cost-effectiveness and long-term patient benefits.

The continued success of voxelotor will depend on its ability to demonstrate sustained clinical value, navigate a competitive and evolving therapeutic landscape, and maintain market access amidst evolving healthcare economics.

Key Takeaways

• Voxelotor (NDC: 42291-0818), marketed as Oxbryta, is a novel oral therapy for sickle cell disease, targeting hemoglobin polymerization.
• The U.S. market for SCD comprises an estimated 100,000 patients, with global prevalence in the millions.
• Voxelotor faces competition from hydroxyurea, crizanlizumab, and emerging gene therapies.
• The drug's list price is approximately $10,000 per month, with net prices varying due to payer negotiations.
• Projected market growth is driven by increased diagnosis, label expansion, and real-world evidence, but restrained by high cost and competition from curative therapies.
• Voxelotor is protected by a portfolio of patents and Orphan Drug Exclusivity, providing market protection until at least late 2026.
• Future outlook includes pediatric expansion, exploration of combination therapies, and strategic integration within Pfizer's global operations.

FAQs

1. What specific patient populations are eligible for voxelotor treatment under the current U.S. indication? Voxelotor is approved for the treatment of sickle cell disease in patients aged four years and older.

2. How does voxelotor's mechanism of action differ from other SCD treatments like hydroxyurea or crizanlizumab? Voxelotor inhibits hemoglobin S polymerization, increasing hemoglobin levels and reducing red blood cell destruction. Hydroxyurea stimulates fetal hemoglobin production, and crizanlizumab blocks P-selectin to reduce VOCs.

3. What is the projected timeline for potential generic entry for voxelotor in the U.S. market? Orphan Drug Exclusivity is in effect until November 2026. Patent expiry is projected around 2030. Generic entry would likely occur after the expiry of both regulatory exclusivity and relevant patents, subject to any legal challenges.

4. Are there any ongoing clinical trials investigating voxelotor for new indications or in combination with other therapies? Yes, Pfizer is conducting research into pediatric expansion and potential combination therapies for voxelotor, with details available through clinical trial registries.

5. What are the primary financial considerations for payers and healthcare systems when evaluating voxelotor? Payers consider the drug's high monthly cost, potential for reduced hospitalizations and complications, long-term disease modification benefits, and comparison to the cost-effectiveness of alternative treatments, including emerging curative therapies.

Citations

1. U.S. Food and Drug Administration. (2019, November 25). FDA approves Oxbryta (voxelotor) tablets to treat sickle cell disease. [Press release]. Retrieved from https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-oxbryta-voxelotor-tablets-treat-sickle-cell-disease
2. Centers for Disease Control and Prevention. (2020, October 22). Sickle Cell Disease. Retrieved from https://www.cdc.gov/ncbddd/sicklecell/data.html
3. World Health Organization. (2021). Sickle-cell anaemia. Retrieved from https://www.who.int/news-room/fact-sheets/detail/sickle-cell-anaemia
4. U.S. Food and Drug Administration. (2019, November 15). FDA approves Adakveo (crizanlizumab-tmqv) to treat sickle cell disease. [Press release]. Retrieved from https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-adakveo-crizanlizumab-tmqv-treat-sickle-cell-disease
5. Global Blood Therapeutics. (2022). Investor Relations Presentations. (Proprietary market analysis data cited in various analyst reports).
6. Pfizer Inc. (2023, February 24). Pfizer’s Oxbryta® (voxelotor) approved by the U.S. Food and Drug Administration as the first and only medicine indicated to treat children ages 4 to 11 years with sickle cell disease. [Press release]. Retrieved from https://www.pfizer.com/news/press-release/press-release-detail/pfizers-oxbrytar-voxelotor-approved-us-food-and-drug
7. GoodRx. (n.d.). Oxbryta prices, coupons, and patient assistance programs. Retrieved from https://www.goodrx.com/oxbryta (Note: Pricing data is an aggregation and may vary).
8. Global Blood Therapeutics. (2022). Form 10-K Annual Report. U.S. Securities and Exchange Commission.
9. U.S. Food and Drug Administration. (2017, July 19). Orphan Drug Designation. Retrieved from https://www.fda.gov/aboutfda/centersoffices/officeofmedicalproductsandtobacco/cder/ucm300021.htm (General information on Orphan Drug Exclusivity, applied to voxelotor upon approval).