The most expensive drug in the world is Lenmeldy, a one-time gene therapy for a rare childhood nerve disease, launched in the United States at $4.25 million per treatment in March 2024. We rank drugs by the US list price (wholesale acquisition cost) the maker set at launch for one treatment, as reported in dated news coverage of each launch. We found no 2025 or 2026 launch above $4.25 million. List prices are what a maker asks, and insurers and health systems often pay less or in instalments.
| Rank | Drug | US launch price | Priced | Treats |
|---|---|---|---|---|
| 1 | Lenmeldy | $4.25 million | Mar 2024 | Metachromatic leukodystrophy |
| 2 (tie) | Hemgenix | $3.5 million | Nov 2022 | Hemophilia B |
| 2 (tie) | Beqvez | $3.5 million | Apr 2024 | Hemophilia B |
| 4 | Elevidys | $3.2 million | Jun 2023 | Duchenne muscular dystrophy |
| 5 | Zevaskyn | $3.1 million | Apr 2025 | Recessive dystrophic epidermolysis bullosa |
1. Lenmeldy: $4.25 million
Lenmeldy treats metachromatic leukodystrophy, a rare inherited nerve disease. It is made from each patient’s own cells. The FDA approved it on Monday 18 March 2024, and the maker, Orchard Therapeutics, announced the $4.25 million list price two days later. It passed Hemgenix to become the highest launch price on record.
How we valued it: wholesale acquisition cost announced by the maker and reported on 20 March 2024. The nonprofit price reviewer ICER had put the treatment’s value at up to about $3.94 million, so the list price came in above that estimate.
2. Hemgenix: $3.5 million
CSL Behring set Hemgenix at $3.5 million when the FDA approved the one-time hemophilia B gene therapy on 22 November 2022, making it the world’s priciest drug at that point. CSL argued the price would cut system costs, since patients have fewer bleeds and need fewer clotting infusions, which can run roughly $550,000 to $750,000 a year.
How we valued it: list price set by the maker at approval, reported 22 November 2022.
2 (tie). Beqvez: $3.5 million
Pfizer matched Hemgenix at $3.5 million for its own one-time hemophilia B gene therapy, with a warranty against treatment failure, when it won approval in late April 2024. Pfizer later discontinued the product, and reports said no patients had received it commercially, so the price is a launch list price only.
How we valued it: launch price reported on 26 April 2024.
4. Elevidys: $3.2 million
Sarepta set the wholesale acquisition cost of its Duchenne gene therapy at $3.2 million after FDA accelerated approval in June 2023. The company said Medicaid and 340B discounts would take the net price roughly 20% below that, and called the price conservative.
How we valued it: price announced by Sarepta’s chief executive on the approval call, June 2023.
5. Zevaskyn: $3.1 million
Abeona Therapeutics priced Zevaskyn, a cell therapy that grafts patient-derived skin sheets onto wounds, at $3.1 million after approval in late April 2025. It comes with an outcomes-based deal that partly refunds the cost if more wound treatment is needed within three years.
How we valued it: list price reported on 30 April 2025.
Close to the top, not ranked
Skysona, bluebird bio’s gene therapy for cerebral adrenoleukodystrophy, launched at about $3 million after its accelerated approval on 16 September 2022. Kebilidi, a PTC Therapeutics gene therapy approved on 13 November 2024, may be priced near the top, but we found no company-confirmed US price and leave it unranked. Zynteglo ($2.8 million, August 2022) and Zolgensma (about $2.1 million, 2019) sit just below.
What drives these prices
- Tiny patient pools. The makers recover costs from a few hundred patients.
- One-time value. The price is set against a lifetime of regular treatment.
- Custom manufacturing. Some therapies are made from each patient’s own cells.
- Payment deals. Makers offer refunds or instalments, so list price and real cost differ.
FAQ
What is the most expensive drug in the world?
Lenmeldy, at a US launch price of $4.25 million for one treatment, announced in March 2024.
What was the most expensive drug before Lenmeldy?
Hemgenix at $3.5 million, set by CSL Behring in November 2022.
Do patients pay these prices?
Rarely in full. Insurers negotiate, and makers often offer refunds or instalment plans.
What does Elevidys cost?
$3.2 million for one treatment, as priced by Sarepta in June 2023.
Is there a more expensive drug in 2026?
We found no launch above $4.25 million in 2025 or 2026. A 2026 industry review of FDA-authorized gene therapies also had Lenmeldy at the top.
How we put this list together
We ranked drugs by the US list price at launch for one course of treatment, from the makers’ announced prices as reported on the day. Prices change, and what patients and insurers pay is usually lower.
Last updated: October 2026.
