Sticker Shock: Check Out the Most Expensive Prescription Drugs in The U.S.
Each year Fierce Pharma, an online news resource, relies upon publicly available data to create an annual Top 10 list of the most expensive medications available in the United States.
The 2025 list was just released and the costs are staggering. Here’s a look:
#1 Lenmeldy. Cost per dose: $4.25 million
Lenmeldy is prescribed to treat a rare genetic disease metachromatic leukodystrophy (MLD), a progressive disorder that damages the central and peripheral nervous systems. The multi-million-dollar drug is rarely prescribed, with reportedly just one U.S. patient starting treatment as of April.
#2 Hemgenix. Cost per dose: $3.5 million
Before Lenmeldy came to market, Hemgenix was the world’s most expensive drug. A gene therapy used to treat hemophilia B, Hemgenix is a one-time treatment for the inherited bleeding disorder. It actually may pencil out as a cost savings, as the drugmaker argues patients with moderate to severe hemophilia B can cost health care systems more than $20 million, over the course of their lives.
#3 Elevidys. Cost per dose: $3.2 million
Elevidys received conditional approval from the FDA in 2023 to treat 4- and 5-year-olds with Duchenne muscular dystrophy (DMD). In 2024, the FDA expanded its approval to cover patients age 4 and older.
#4 Lyfgenia. Cost per dose: $3.1 million
The drug is approved for patients with sickle cell disease and a history of vaso-occlusive events, which can damage vital organs.
#5 Skysona. Cost per dose: $3 million.
The medication is a one-time treatment for boys ages 4 to 17 with early and active cerebral adrenoleukodystrophy, a progressive neurological disease that can lead to complete loss of voluntary movement. Absent any treatment, about half of patients typically die within five years of experiencing the first symptoms. An estimated one in 21,000 newborn males in the U.S. are affected by the disease and about 40 patients in the U.S. could be eligible for the drug each year.
#6 Roctavian. Cost per dose: $2.9 million
Roctavian is also a one-time gene therapy prescribed to treat the bleeding disorder, hemophilia B. The manufacturer offers a partial reimbursement if the drug’s effects begin to wane within the first four years after dosing.
#7 Rethymic. Cost per dose: $2.81 million
The drug is prescribed to recover immune function in children with congenital athymia, an ultra-rare immunodeficiency disease. Those with the disease are born without a functioning thymus gland. Without targeted treatment, people born with the disease typically live just two to three years. It is extremely rare in the U.S., occurring in about 17 to 24 of the millions of U.S. births per year.
#8 Zynteglo. Cost per dose: $2.8 million
Zynteglo was among the first class of highly expensive gene therapies, first approved for use in 2022. It is used to treat beta thalassemia, a condition that requires regular blood cell transfusions. According to the manufacturer people who live with beta thalassemia can rack up medical costs of up to $6.4 million in their lifetimes. The eligible patient pool is estimated to number 1,300 to 1,500 people in the U.S.
#9 Zolgensma. Cost per dose: $2.32 million
This is a one-time treatment used to treat spinal muscular atrophy by administering the genetic material directly into the spinal cord.
#10 Casgevy. Cost per dose: $2.2 million
Rounding out the tenth spot, is Casgevy, a gene therapy developed to treat sickle cell disease. It was the first medicine using the revolutionary CRISPR gene editing system that allows scientists to modify DNA sequences within living cells. According to Fierce Pharma, many patients don’t pursue gene therapy because there are other effective treatments, including blood transfusions and oral medication that has been found to be effective (and likely, much less expensive.)
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