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Ranked: America’s Most Expensive Drugs
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Key Takeaways
- Lenmeldy is America’s priciest drug in 2025 at $4.25 million per dose.
- One Lenmeldy treatment equals roughly 12,500 Ozempic doses ($342 each, before insurance)
In 2025, the soaring cost of cutting-edge gene therapies has pushed individual drug prices to record highs.
The latest ranking of America’s most expensive drugs highlights how a single treatment can rival the price of a luxury home.
The data for this visualization comes from Fierce Pharma. It lists the 10 priciest U.S. drugs, all topping $2 million per course and most offering one-time, potentially curative benefits
Gene Therapies Dominate the Leaderboard
Lenmeldy, a treatment for the ultrarare disorder metachromatic leukodystrophy, costs $4.25 million per dose, eclipsing every other therapy launched to date.
| Rank | Drug Name | Cost Per Dose | Company | Used For |
|---|---|---|---|---|
| 1 | Lenmeldy | $4,250,000 | Kyowa Kirin | A gene therapy used to treat kids with metachromatic leukodystrophy (MLD), a rare inherited metabolic disorder |
| 2 | Kebilidi | $3,950,000 | PTC Therapeutics | A gene therapy used to treat children & adults with AADC deficiency, a rare disorder that prevents the body from making key brain chemicals |
| 3 | Hemgenix | $3,500,000 | CSL Behring | A one-time gene therapy used to treat adults with hemophilia B to reduce bleeding episodes |
| 4 | Elevidys | $3,200,000 | Sarepta Therapeutics | A gene therapy used to treat Duchenne muscular dystrophy (DMD) in people 4 years and older |
| 5 | Lyfgenia | $3,100,000 | bluebird bio | A one-time gene therapy used to treat sickle cell disease with a history of pain crises |
| 6 | Skysona | $3,000,000 | bluebird bio | A gene therapy used to slow nerve damage in boys with early, active cerebral adrenoleukodystrophy (CALD) |
| 7 | Roctavian | $2,900,000 | BioMarin | A one-time gene therapy used to treat adults with severe hemophilia A who don’t have AAV5 antibodies |
| 8 | Rethymic | $2,810,000 | Sumitomo Pharma | A tissue-based therapy used to help kids with congenital athymia build a working immune system |
| 9 | Zynteglo | $2,800,000 | bluebird bio | A gene therapy used to treat people with transfusion dependent beta thalassemia |
| 10 | Zolgensma | $2,320,000 | Novartis | A one-time gene therapy used to treat children under 2 with spinal muscular atrophy (SMA) |
Note: Bluebird Bio is now Genetix Biotherapeutics after acquisition by two private equity firms.
Lenmeldy’s list price equals roughly 12,500 doses of popular diabetes drug Ozempic at its pre-insurance list price.
While the number seems astronomical, payers weigh it against lifelong care costs that can exceed $10 million for untreated MLD patients.
Similarly, third-ranked Hemgenix’s one-time $3.5 million cost compares with up to $20 million for decades of clotting-factor infusions.
Even at multimillion-dollar stickers, pay-once gene therapies can offer health-economic value over chronic treatments.
In fact, every drug on the top 10 list is a gene or cell-based therapy—scientific breakthroughs that replace or repair faulty genetic instructions.
Because they aim to cure rare and deadly conditions in a single dose, their development and manufacturing pipelines are complex, bespoke, and expensive.
Related: Check out where Ozempic ranks in America’s most common drugs by medicare spending.
Bluebird Bio’s Three-Drug Footprint
No company appears more often than Bluebird Bio, which places Lyfgenia, Skysona, and Zynteglo on the list.
Each addresses a different inherited blood or metabolic disorder, yet all share core technology roots developed over a decade.
Despite regulatory scrutiny and manufacturing setbacks, the company’s persistence has translated into multiple FDA approvals.
The cluster illustrates how a single firm can dominate a high-value therapeutic niche.
Bluebird Bio was acquired in June, 2025 by private equity firms Carlyle Group and SK Capital.
Learn More on the Voronoi App
For related coverage, check out Where Americans Pay the Most (and Least) for Health Insurance on Voronoi, the new app from Visual Capitalist.