Last updated: February 24, 2026
What is the Drug Associated with NDC 31722-0749?
NDC 31722-0749 refers to a lysosomal acid lipase (LAL) deficiency treatment, likely Sebelipase alfa (brand name Kanuma). Kanuma is an enzyme replacement therapy approved for LAL deficiency, a rare autosomal recessive disorder affecting lipid metabolism.
Current Market Position
Sebelipase alfa entered the market in 2015, with initial pricing established at approximately $300,000 annually per patient. It is marketed solely by Alexion Pharmaceuticals (a subsidiary of AstraZeneca). As of 2022, the approved indications include lysosomal acid lipase deficiency in both pediatric and adult populations.
Market Size and Incidence
- Disease prevalence: Estimated at 1 in 300,000 to 1 in 400,000 globally.
- US prevalence: Approximate annual diagnosed cases are 200-400.
- Potential addressable population: 600-800 in the US, with similar ratios globally.
Competitive Landscape
Limited, as no alternative enzyme replacement therapies treat LAL deficiency directly.
| Drug Name |
Market Entry Year |
Approval Status |
Price (Approximate) |
Notable Competitors |
| Sebelipase alfa (Kanuma) |
2015 |
Approved |
$300,000/year |
None direct |
Market Trends and Limitations
- Demand drivers: Increased diagnosis via genetic testing.
- Economic barriers: High pricing restricts access; payor resistance exists.
- Patient burden: Chronic, lifelong treatment needed.
Pricing Trends and Projections
Historical Pricing
- Initial listing at approximately $300,000/year in 2015.
- Slight adjustments over subsequent years, with some estimates indicating increases of 2-4% annually driven by inflation and healthcare inflation trends.
Projected Pricing (2023-2028)
| Year |
Estimated Price |
Rationale |
| 2023 |
$310,000 |
3-4% inflation adjustment |
| 2024 |
$320,000 |
Continued inflation, potential payer negotiations |
| 2025 |
$330,000 |
Adjustments for market and demand shifts |
| 2026 |
$340,000 |
Market stabilization but inflation persists |
| 2027 |
$350,000 |
Slight premium on higher diagnosis rates |
Market Factors Impacting Price
- Expansion in diagnosed patient pool.
- Negotiations and discounts offered by payors.
- Entry of biosimilar or alternative treatments (none currently exists).
- Regulatory policy shifts influencing drug pricing transparency.
Market Dynamics and Future Opportunities
- Potential expansion: Pediatric extension or early intervention could increase demand, raising prices.
- Regulatory landscape: Emerging policies to control high-cost drug prices could pressure future pricing.
- Research pipeline: No approved biosimilars or generics yet, maintaining high barriers to entry for competitors.
Key Market Risks
- Pricing pressures: Managed markets progressively scrutinize high-cost therapies.
- Market size limitations: Rare disease prevalence constrains revenue growth.
- Reimbursement challenges: Payer resistance may limit access, affecting sales volume.
Summary of Market Outlook
- The market for NDC 31722-0749 (Sebelipase alfa) is stable with minimal competition.
- Pricing is expected to increase modestly, reflecting inflation and demand growth.
- High development, manufacturing costs sustain premium pricing.
- Expansion in diagnosed populations and treatment indications could elevate both sales volume and revenue.
Key Takeaways
- The drug's high cost remains a barrier despite increasing demand.
- Market expansion hinges on better diagnosis rates and broader indications.
- Pricing projections suggest modest annual increases of approximately 3-4%.
- Competition is limited; biosimilar threat remains distant.
- Payer negotiations and policy changes will influence future pricing dynamics.
FAQs
1. What factors most influence the drug’s pricing?
Demand size, manufacturing costs, regulatory policies, and payer negotiations primarily drive pricing.
2. How might biosimilars impact market price?
Biosimilars could emerge within 5-7 years but are unlikely to significantly reduce prices soon given the rarity of the condition and current lack of competitors.
3. What is the global market size for this drug?
Estimated at 600-800 patients globally, constrained by the disease’s rarity.
4. Will expanded indications improve sales volume?
Yes, expanding use to younger populations or earlier stages could increase demand but may not significantly impact price.
5. How could policy changes influence future price projections?
Policies targeting high-cost drugs could cap reimbursement levels, delaying price increases and possibly leading to discounts.
References
- National Organization for Rare Disorders (NORD). (2022). Lysosomal Acid Lipase Deficiency. https://rarediseases.org/rare-diseases/lysosomal-acid-lipase-deficiency/
- AstraZeneca. (2022). Kanuma (Sebelipase alfa) Product Label.
- IQVIA. (2023). U.S. Pharmaceutical Price Report.
- EvaluatePharma. (2022). World Market for Enzyme Replacement Therapies.
- CMS. (2023). Policy on Drug Pricing Transparency and Reimbursement.
