The cost of prescription drugs has long been a hot-button issue in healthcare, but some therapies come with price tags so high they seem unreal. These treatments, many of which are for rare diseases or genetic conditions, often cost millions per year—or even for a one-time treatment.
Below is a list of the 20 most expensive drugs based on their list price in the U.S. healthcare market, including gene therapies and life-saving innovations approved by the FDA. Prices are for a single course or per year, depending on the treatment type.
Table of Contents
Top 20 Most Expensive Drugs (2024)
| Rank | Drug Name | List Price (USD) | Use / Condition |
| 1 | Zolgensma (Novartis) | $2.25 million (one-time) | Spinal Muscular Atrophy (SMA) – genetic muscle weakness in infants |
| 2 | Hemgenix (CSL Behring) | $3.5 million (one-time) | Hemophilia B – blood clotting disorder due to Factor IX deficiency |
| 3 | Skysona (Bluebird Bio) | $3.0 million (one-time) | Cerebral Adrenoleukodystrophy (CALD) – fatal brain disorder in boys |
| 4 | Zynteglo (Bluebird Bio) | $2.8 million (one-time) | Beta-thalassemia – genetic anemia requiring red blood cell transfusions |
| 5 | Elevidys (Sarepta Therapeutics) | $3.2 million (one-time) | Duchenne Muscular Dystrophy (DMD) – progressive muscle weakness |
| 6 | Lenmeldy (Orchard Therapeutics) | $4.25 million (one-time) | Metachromatic Leukodystrophy (MLD) – affects nervous system and movement |
| 7 | Luxturna (Spark Therapeutics) | $850,000 (one-time) | Genetic blindness caused by RPE65 mutation |
| 8 | Zokinvy (Eiger BioPharmaceuticals) | $1.1 million/year | Hutchinson-Gilford Progeria Syndrome – premature aging disorder |
| 9 | Myalept (Amryt) | $1.0 million/year | Lipodystrophy – rare fat metabolism disorder treated with leptin |
| 10 | Danyelza (Y-mAbs Therapeutics) | $977,664/year | Neuroblastoma – rare pediatric cancer |
| 11 | Kimmtrak (Immunocore) | $975,520/year | Metastatic Uveal Melanoma – eye cancer |
| 12 | Carvykti (Janssen/Legend Biotech) | $465,000 (one-time) | Multiple Myeloma – blood cancer |
| 13 | Breyanzi (Bristol Myers Squibb) | $410,300 (one-time) | Lymphoma – immune system cancer |
| 14 | Yescarta (Kite Pharma/Gilead) | $399,000 (one-time) | Large B-cell lymphoma |
| 15 | Tecartus (Kite Pharma/Gilead) | $424,000 (one-time) | Mantle cell lymphoma |
| 16 | Adcetris (Seagen) | $350,000/year | Hodgkin’s lymphoma and T-cell lymphomas |
| 17 | Blincyto (Amgen) | $350,000/year | Acute lymphoblastic leukemia |
| 18 | Folotyn (Acrotech Biopharma) | $320,000/year | Peripheral T-cell lymphoma |
| 19 | Vimizim (BioMarin) | $300,000–$500,000/year | Morquio A syndrome – enzyme replacement for rare genetic disorder |
| 20 | Soliris (Alexion/AstraZeneca) | $500,000/year | Paroxysmal Nocturnal Hemoglobinuria (PNH) and aHUS |
Why Are These Drugs So Expensive?
1. Rare Disease Treatments
Most of these drugs are approved for ultra-rare genetic diseases, affecting very small patient populations. This limits the ability of pharmaceutical companies to recoup development costs through volume.
2. Gene Therapy & One-Time Treatments
Cutting-edge gene therapies like Zolgensma, Zynteglo, and Lenmeldy offer potential cures with a single dose, often targeting disorders at the DNA level—especially in children under a few years of age.
3. FDA Approval and Breakthrough Designation
Many of these therapies received accelerated approval or breakthrough therapy designation, allowing for faster development and higher initial price tags.
4. Manufacturing Complexity
Treatments involving bone marrow, red blood cells, or immune system engineering require highly individualized, labor-intensive processes.
Who Pays for These Drugs?
- Medicare, Medicaid, and private insurance cover some costs, but copay amounts can still be devastating.
- Many companies offer financial assistance or patient support programs.
- Some drugs are part of cost-saving value-based contracts (e.g., “only pay if it works”).
Controversy and Ethics
High drug prices raise ethical questions about access to life-saving care, healthcare equity, and profit models in the pharma industry. While these drugs often represent medical breakthroughs, they also highlight the tension between innovation and affordability.
Final Thoughts
These most expensive drugs reflect the future of precision medicine, especially for rare conditions and genetic diseases. While their price tags can be staggering, they often offer hope where none existed before.
Still, the rising cost of brand-name prescription drugs in the U.S. continues to challenge healthcare systems, insurers, and patients alike.
If you or a loved one may benefit from one of these treatments, speak to a healthcare provider or treatment program about eligibility, access, and financial support.
Published on: 2025-06-04
Updated on: 2025-06-04
