Last updated: February 27, 2026
What is NDC 42794-0005?
NDC 42794-0005 refers to a specific drug product listed in the National Drug Code directory. Based on available data, this NDC corresponds to Sebelipase alfa (Kanuma), a recombinant enzyme indicated for the treatment of lysosomal acid lipase deficiency (LAL-D).
Market Size and Demand
Patient Population
LAL-D is a rare genetic disorder with an estimated prevalence of approximately 2 to 4 per million live births. The primary target population is:
- Infants and children with Wolman disease and persistent dyslipidemia.
- Adults with rapidly progressive non-alcoholic fatty liver disease due to LAL-D.
Estimated Patient Numbers
Based on U.S. and global prevalence data:
| Population Segment |
Estimated Patients (Global) |
Estimated Patients (U.S.) |
| Pediatric LAL-D cases |
200-500 |
40-100 |
| Adult LAL-D cases |
500-1,000 |
100-200 |
The total addressed market could be approximately 700 to 1,500 patients globally.
Market Drivers
- FDA approval for specific indications.
- Limited competition; only one other enzyme replacement therapy (alglucerase) for similar indications.
- High unmet medical need due to disease severity and lack of alternative therapies.
Key Market Trends
- Growing awareness and diagnosis of LAL-D.
- Expansion of indications, with ongoing clinical trials for broader age groups.
- Reimbursement landscape stabilizes with payer acceptance.
Pricing Dynamics
Current Pricing Structure
As of 2023, Kanuma’s wholesale acquisition cost (WAC) approximates $600,000 to $700,000 annually per patient in the U.S. The high cost stems from:
- The complex manufacturing process for recombinant enzymes.
- Rare disease designation allowing premium pricing.
- Administration requirements (intravenous infusion).
Price Comparisons
- Sebelipase alfa (Kanuma): ~$600,000 - $700,000/year.
- Other enzyme replacement therapies (e.g., Fabrazyme for Fabry disease): similar pricing ranges ($500,000 - $700,000/year).
- Off-label and experimental treatments tend to be significantly lower, but are not approved or reimbursed.
Market Share Projections
- Current market penetration remains limited to specialized centers.
- Adoption expected to grow with increased diagnosis and expanded indications.
- Price remains relatively stable due to lack of generic or biosimilar competition, though biosimilar development is a future possibility.
Price Projections (2023–2028)
| Year |
Projected Price Range (Annual) |
Notes |
| 2023 |
$600,000 - $700,000 |
Current market price |
| 2024 |
$590,000 - $690,000 |
Slight discounts expected with market expansion |
| 2025 |
$580,000 - $680,000 |
Volume-driven discounts possible |
| 2026 |
$570,000 - $670,000 |
Potential payer negotiations |
| 2027 |
$560,000 - $660,000 |
Competitive pressures may arise |
| 2028 |
$550,000 - $650,000 |
Biosimilar entries possible |
Key assumptions:
- Stable manufacturing costs.
- No significant regulatory changes altering pricing.
- Continued payer acceptance driven by clinical necessity.
Regulatory and Market Risks
- Biosimilar competition could lower prices.
- Reimbursement policies may tighten for high-cost therapies.
- Expanding indications can increase sales volume but may pressure price points.
Key Takeaways
- NDC 42794-0005 (Sebelipase alfa/Kanuma) addresses a rare but serious genetic disorder, with a dosing cost approaching $600,000/year.
- The market remains limited but has potential for growth via increased diagnosis, expanded indications, and payer acceptance.
- Price stability is expected over the next five years with potential decline due to biosimilars.
- Few competitors exist, supporting positioning as a premium-priced therapy in its niche.
- Market expansion depends on clinical progress, regulatory decisions, and payer negotiations.
FAQs
1. How does the price of NDC 42794-0005 compare to other rare disease treatments?
It falls within the typical range of $500,000 to $700,000 annually, similar to therapies like Fabrazyme for Fabry disease, due to manufacturing complexity and limited market size.
2. What factors could lead to price reductions?
Biosimilar development, increased competition, and payer negotiations could lower prices. Healthcare policy shifts aiming at cost containment also influence pricing.
3. What are the main clinical hurdles for expanding the use of this drug?
Demonstrating efficacy in broader patient populations and obtaining regulatory approval for expanded indications are primary challenges.
4. How does market demand influence pricing?
Higher patient identification and a broader indication spectrum may increase volume, but price per unit might decline to accommodate payer constraints without reducing revenue.
5. What is the outlook for biosimilar competition?
Biosimilar development is in early stages; approval and market entry could occur in 5-10 years, potentially impacting pricing and market share.
References
[1] U.S. Food and Drug Administration. (2015). Kanuma (Sebelipase alfa) approval letter.
[2] IQVIA. (2023). Pharmaceutical Price and Market Data.
[3] GlobalData. (2023). Rare Disease Market Analysis.
[4] National Organization for Rare Disorders. (2022). Lysosomal Acid Lipase Deficiency.
[5] FDA. (2022). Guidance for Industry: Biosimilar Product Development.
