Last updated: February 21, 2026
What is the Clinical and Therapeutic Profile of NDC 42543-0962?
NDC 42543-0962 corresponds to the drug Empaveli (pegcetacoplan), a complement C3 inhibitor targeted for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Approved by the FDA in July 2021, Empaveli offers an alternative to existing complement pathway inhibitors such as Soliris (eculizumab) and Ultomiris (ravulizumab).
Key characteristics:
- Indication: PNH, a rare hematologic disorder
- Administration: Subcutaneous injection
- Pricing: Priced at approximately $519,118 annually based on current estimates (2023)
Market Landscape and Competitive Position
Market Size and Patient Population
The PNH global market has expanded with increased awareness and diagnosis rates, estimated to reach approximately 4,500 patients in the U.S. alone. Historically, the market was dominated by Soliris, with annual sales exceeding $4 billion (Pharmaceutical Business Review, 2022). Ultomiris has captured a market share due to dosing convenience.
Competitive Dynamics
| Drug |
Approval Year |
Route |
Annual Sales (2022) |
Market Share |
Price (per year) |
| Soliris (eculizumab) |
2007 |
IV infusion |
$4.3 billion |
70% |
$678,000 |
| Ultomiris (ravulizumab) |
2018 |
IV infusion |
$1.2 billion |
25% |
$609,000 |
| Empaveli (pegcetacoplan) |
2021 |
Subcutaneous |
Limited data; initial indications |
5% |
$519,118 |
Empaveli’s subcutaneous administration improves patient convenience but is priced lower than IV counterparts, reflecting a competitive positioning to penetrate the market.
Price Trends and Projection Drivers
Historical Pricing Trends
- Soliris: Maintained high prices since 2007. Over the last decade, annual increases hovered around 8-10% annually.
- Ultomiris: Introduced at a slight discount, with initial pricing at $609,000 in 2018; prices increased modestly in subsequent years.
- Empaveli: Priced at $519,118 upon launch, roughly 24% less than Soliris, aiming for market penetration.
Projected Price Trajectory (2023-2028)
| Year |
Estimated Price (USD) |
Rationale |
| 2023 |
$519,118 |
Stable at launch; market negotiations could influence discounts |
| 2024 |
$520,500 |
Slight inflation, consistent with historical trends |
| 2025 |
$525,000 |
Possible discounts for increased market penetration |
| 2026 |
$530,000 |
Slight increase, market acceptance improves |
| 2027 |
$535,000 |
Potential competition-induced price stabilization |
| 2028 |
$540,000 |
Price plateau as market matures |
Factors Influencing Price Changes
- Patent exclusivity: Pending expiration timelines can pressure pricing.
- Market competition: Introduction of biosimilars or innovative therapies could compel price reductions.
- Reimbursement policies: Value-based reimbursement models may favor discounts for improved convenience or efficacy.
- Manufacturing costs: Technology enhancement or scale effects could reduce costs over time, enabling lower pricing.
Market Entry and Adoption Factors
- Physician preference: Subcutaneous administration drives adoption among patients preferring home-based therapy.
- Regulatory developments: Expansion beyond PNH to other complement-mediated disorders can increase market size.
- Pricing strategies: Market penetration often involves initial discounts; long-term prices align with perceived value and competitive pressures.
Regulatory and Market Access Environment
- PBMs and insurers assess value relative to existing therapies.
- Cost-effectiveness analyses support pricing negotiations.
- The Orphan Drug designation provides incentives but does not restrict pricing strategies.
Key Takeaways
- Empaveli is positioned to capture incremental market share through convenience and pricing that undercuts IV alternatives.
- Pricing remains high, but expected to increase modestly over the next five years.
- Market growth depends on expanding indications, competitive dynamics, and payer acceptance.
- The current annual price is approximately $519,118, with projections reaching near $540,000 by 2028.
FAQs
1. How does Empaveli pricing compare to Soliris and Ultomiris?
Empaveli’s initial price is approximately 23-24% lower than Soliris and Ultomiris. Over time, prices may approach those of IV therapies due to market and reimbursement pressures.
2. What factors could lead to significant price reductions?
Entry of biosimilars, changes in patent status, or value-based pricing models could reduce prices. Market competition generally exerts downward pressure.
3. How has the market responded to Empaveli since its approval?
Early adoption is focused on patients seeking subcutaneous options; full market penetration remains limited due to established therapies’ dominance.
4. What is the potential for Empaveli in other indications?
Research is ongoing to evaluate its efficacy for hemolytic syndromes and other complement-mediated disorders, which could expand its market.
5. What is the likelihood of pricing stabilization by 2028?
Prices are expected to stabilize as the market matures, with moderate increases aligned with inflation and value perception rather than aggressive hikes.
References
- Pharmaceutical Business Review. (2022). Market analysis of PNH therapies.
- U.S. Food and Drug Administration. (2021). Empaveli (pegcetacoplan) approval documentation.
- IQVIA. (2023). Global Oncology and Hematology drug market estimates.
- Institute for Clinical and Economic Review. (2022). PNH therapies comparative value assessment.
- CNBC. (2022). Drug pricing trends and market dynamics.
Note: Price estimates are based on publicly available data as of 2023 and may vary with negotiations, new indications, or market developments.
