Last updated: February 23, 2026
What is NDC 42291-0475?
NDC 42291-0475 corresponds to Pemigatinib, marketed as Pemazyre®, a targeted therapeutic approved for certain types of cholangiocarcinoma and other cancers involving FGFR alterations.
Approved Indications and Market Size
Pemigatinib gained FDA approval in April 2020 for previously treated, unresectable or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. As of 2023, its regulatory scope expanded to include additional tumor types with FGFR alterations.
Patient Population
- Cholangiocarcinoma (bile duct cancer): Approximately 8,000 new cases annually in the U.S.
- FGFR2 fusions/rearrangements occur in 10-15% of intrahepatic cholangiocarcinoma cases.
- The prevalence of FGFR alterations in other solid tumors is lower but growing as testing broadens.
Market Penetration
- In 2022, Pemazyre sales surpassed $100 million globally, reflecting rapid uptake in oncology centers testing for FGFR mutations.
- A key driver remains the increasing adoption of molecular testing.
Competitive Landscape
Direct Competitors
- Infigratinib (QED-229): Approved for FGFR-driven cholangiocarcinoma.
- Futibatinib (Tresoso™): Approved for FGFR2 fusion-positive cholangiocarcinoma.
- Erdafitinib (Balversa): Approved for FGFR-mutant bladder cancer.
- These narrow indications limit market overlap but signify a growing class of FGFR inhibitors.
Indirect Competitors
- Chemotherapy options with broader indications.
- Emerging targeted therapies in early development.
Market Drivers
- Increasing use of next-generation sequencing (NGS) panels.
- Growing number of FGFR-positive tumor diagnoses.
- Expanded indications and label extensions.
Future Approvals
- Trials expanding Pemigatinib's use in other FGFR-driven cancers.
- Potential approval for earlier lines of therapy if phase 3 data is positive.
Price Projections and Revenue Outlook
Current Pricing
- Average wholesale price (AWP): Approximately $14,000 per month per patient.
- Treatment duration: Typically 6-12 months, depending on response.
Revenue Estimates
| Year |
Estimated Patients (U.S.) |
Estimated Revenue |
Comments |
| 2023 |
5,000 |
~$840 million |
Growth driven by increased testing and approval breadth. |
| 2025 |
8,000 |
~$1.3 billion |
Market expansion, higher adoption, label extensions. |
| 2030 |
15,000 |
~$2.8 billion |
Potential approval for additional indications. |
Price Trend Factors
- Biosimilar competition unlikely soon due to small molecule nature.
- Price may decrease incrementally as competition enters.
- Payer negotiations could influence net pricing; current high prices remain based on targeted therapy value and rarity.
Influencing Factors on Price Trajectory
- Expanded indications may justify maintaining or increasing prices.
- Increased volume may compensate for price reductions.
- Developing markets and healthcare system differences could alter cost structures regionally.
Risks and Challenges
- Entry of competitors with superior efficacy or safety profiles.
- Rapid adoption of multiplex testing may dilute per-drug pricing.
- Patent expiry scheduled for late 2020s, risking generic erosion.
Policy and Regulatory Outlook
- Emphasis on personalized medicine and molecular testing supports continued demand.
- Potential for accelerated approvals or expanded label indications based on ongoing trials.
- Pricing pressure from payers and healthcare systems seeking value-based models.
Key Takeaways
- Pemigatinib remains a high-growth targeted therapy fueled by increasing FGFR testing.
- Current price remains around $14,000/month, with revenue projections reaching ~$2.8 billion globally by 2030.
- Competitive landscape is emerging, but Pemigatinib's specific indications and molecular targeting support sustained demand.
- Market expansion hinges on broader testing and regulatory approvals.
- Price reductions are possible but marginal given the rarity and specificity of the indications.
FAQs
1. What factors most influence Pemigatinib's future pricing?
Market expansion, competition, regulatory changes, and healthcare policy shifts significantly impact pricing.
2. How does molecular testing affect Pemigatinib's market penetration?
Broader testing increases diagnosed cases, directly boosting patient access and sales.
3. What is the expected timeline for biosimilar entry?
Biosimilar competition for small molecule targeted therapies is unlikely before late 2020s due to patent protections.
4. Are there plans to expand Pemigatinib's indications?
Clinical trials are ongoing for additional FGFR-driven cancers, which could broaden the drug's use.
5. How does Pemigatinib's pricing compare to similar targeted agents?
Pricing is comparable to other FGFR inhibitors, typically around $14,000/month, reflecting the targeted therapy premium.
References
[1] U.S. Food and Drug Administration. (2020). FDA approves Pemigatinib for cholangiocarcinoma. Retrieved from https://www.fda.gov
[2] IQVIA. (2023). Oncology drug sales and market data.
[3] EvaluatePharma. (2023). Oncology market projections.
