Key takeaways:
Zolgensma is the most expensive drug in America, with an average one-time cost of $2.1 million.
Since September, six of the most expensive medications in the U.S. have seen a price increase.
Kimmtrak, a newly approved drug, is the most recent medication to make the list.
Many drugs are expensive — but some drugs are wildly expensive, according to a recent analysis by GoodRx Research.
The GoodRx Research team regularly tracks the most expensive medications filled at local pharmacies in the U.S., and drugs on that list have only gotten more expensive since we began tracking their prices. The list’s top 3 drugs — Zokinvy, Myalept, and Mavenclad — all cost over $60,000 for a typical monthly supply. However, these are the most expensive drugs filled at pharmacies, and patients self-administer them on a regular basis.
With more approvals of expensive infusions, cancer treatments, and gene therapies, we wanted to track the most expensive drugs, including those not available from a pharmacy. We call these drugs “healthcare provider-administered drugs,” because only a medical professional can administer them.
Many of the most expensive drugs are taken for an extended period of time, and costs quickly add up. This analysis ranks drugs by their annual cost for a typical course of therapy. Prices are based on the drug’s list price — which is the price that the pharmaceutical company assigns as an official price to a drug — then adjusted for the annual timeframe to create a comparable benchmark.
The following are the 10 most expensive drugs in the U.S., taking into account both drugs that you can get at a pharmacy and healthcare provider-administered drugs. Relatively few people take any of these drugs — but the outsized costs can translate into higher premiums and more restrictive formularies for everyone.
1. Zolgensma – $2,125,000
Zolgensma received FDA approval in May 2019. It has remained the most expensive drug in the U.S., with a one-time cost of $2.1 million for a course of treatment.
Zolgensma is approved to treat spinal muscular atrophy, a rare childhood disorder that causes muscular erosion that can lead to lung infections and muscle weakness. Zolgensma is a one-time curative therapy, and many children who have been treated with the drug now show no signs of the disease.
Paying for Zolgensma isn’t easy, as insurers may not cover it or may have certain requirements in order for people to qualify for coverage. To encourage insurers to cover this gene therapy, manufacturer Novartis Gene Therapies has allowed certain insurers to pay for the drug in annual installments of $425,000 over 5 years.
Novartis Gene Therapies also offers the OneGene Program to provide guidance and help navigate insurance requirements.
2. Zokinvy – $1,073,760
Zokinvy, an orphan drug, was approved by the FDA in November 2020. Its cost is staggering, at over $1 million a year. It is the first and only treatment approved for Hutchinson-Gilford progeria syndrome, a rare genetic disease that causes premature aging.
Although the dosage depends on body surface area, people typically take around 200 mg of Zokinvy a day, and, at $746 per 50 mg capsule, this adds up to $89,480 a month. This cost reflects a 4% increase that occurred in December 2021.
While there is no copay card available for this drug, the Eiger OneCare program can help assist with insurance coverage and financial support options.
3. Danyelza – $1,011,882
The FDA approved Danyelza in November 2020 to treat neuroblastoma in the bone or bone marrow of pediatric and adult patients. Danyelza was under an accelerated approval program and is considered an orphan drug and breakthrough therapy.
However, the medication is not cheap. In December 2021, Danyelza increased in price 3.5%, bringing the cost of a single vial to $21,081. People typically use around 48 vials per year, bringing the annual cost close to $1,011,882.
Manufacturer Y-mAbs Therapeutics offers Y-mAbs Connect, a program that can help people navigate insurance, as well as a patient assistance program for eligible people without insurance.
4. Kimmtrak – $975,520
The newest drug to make the list, Kimmtrak was approved by the FDA in January 2022 to treat metastatic or surgically untreatable uveal melanoma, a form of eye cancer. Kimmtrak is administered via IV infusion by a healthcare provider, and the typical person will use one vial per week, every week, as a maintenance dosage. A single vial costs $18,760, which brings the yearly cost of treatment to $975,520.
Manufacturer Immunocore Ltd offers opportunities for qualified patients to save through their KimmtrakConnect financial assistance and patient support program.
5. Myalept – $929,951
Myalept is one of the only drugs on this list that is dispensed at the pharmacy. It is used to treat leptin deficiency in patients with generalized lipodystrophy, which causes abnormal fat distribution in the body.
Myalept costs $77,496 for a monthly supply. As of January 2022, this price is up 4.5% from our last September update. People self-administer Myalept once a day and typically use 14 vials per month, bringing the yearly price tag to $929,951. Because Myalept is the only treatment available to control this rare condition, there are no other cost-saving alternatives.
Manufacturer Amryt Pharmaceuticals offers assistance programs to help some people afford Myalept.
6. Luxturna – $850,000
At $850,000 a year, Luxturna is the sixth-most expensive drug on the list. Luxturna is a gene therapy that treats an inherited form of retinal dystrophy, a condition that causes vision loss and often even complete blindness.
Luxturna cannot be dispensed at a pharmacy, as it requires a doctor to administer 1 vial of the medication into each eye. People should only need one dose of Luxturna ever, but the list price is steep, ringing up at $425,000 per vial.
Spark Therapeutics Generation Patient Services can provide assistance by helping to navigate insurance coverage and connecting patients to other financial resources.
7. Folotyn – $842,585
Folotyn is approved to treat peripheral T-cell lymphoma, a rare blood cancer that can cause death. A healthcare professional must administer Folotyn, and people are typically given 135 vials of the drug annually. Manufacturer Acrotech Biopharma increased the list price for a vial by 3% in January 2022 — from $6,058 to $6,241 — bringing the total annual cost to $842,585.
However, the manufacturer does offer savings opportunities to qualified patients through their STAR program, which can help lower this cost.
8. Brineura – $755,898
Brineura is a first-in-class medication that treats a specific form of Batten disease called late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Specifically, Brineura helps treat the slow loss of walking ability in pediatric patients 3 years and older.
Following a price increase of 3.5% this January, the list price of Brineura is now $29,073 for a kit of 2 vials of 150 mg. The recommended dosage is 300 mg every 2 weeks, meaning that over the course of one year, costs add up to $755,898.
Manufacturer BioMarin does provide financial support for insured and cash-paying patients through their copay assistance program.
9) Blincyto – $754,720
The ninth-most expensive drug, Blincyto, has an annual list price of $754,720. Manufactured by Amgen, Blincyto is used to treat a rare form of acute lymphoblastic leukemia, a cancer of the blood and bone marrow.
Only a healthcare provider can administer Blincyto, and dosing is done in cycles. The first cycle — known as the induction phase — is aimed at reducing the number of cancer cells. Cycles two through four — known as consolidation phases — help new healthy cells grow. People need different amounts of the medication during each phase but will typically use about 168 vials per year. The current list price is $4,492 per vial following a price increase of 5.9% that occurred in January 2022.
Amgen offers a way for some people to lower the monthly cost of Blincyto through their Safety Net Foundation.
10. Ravicti – $695,970
Used to treat urea cycle disorders, Ravicti has an annual list price of $695,970. Urea cycle disorders are genetic conditions that result in high levels of ammonia in the blood. If left untreated, they can lead to confusion, coma, or even death.
Ravicti is dispensed in a pharmacy and doesn’t require administration from a healthcare provider. People are typically given 132 bottles annually. The list price of Ravicti is $5,273 for one bottle.
Manufacturer Horizon Therapeutics offers a way for some patients to save on Ravicti through the Horizon Cares Patient Assistance Program.
Co-contributors: Jeroen van Meijgaard, PhD, Jennifer Tran, PharmD, Lauren Chase, and Tori Marsh, MPH
Methodology
We created a list of the most expensive drugs by list price, including drugs that are administered under the supervision of a healthcare provider, and obtained typical fill quantities using data and information from the drug manufacturer websites. Using the official manufacturer list price, we calculated the total annual cost of a typical course of therapy for each drug. For drugs where there is a large effective dosing range, or where patient weight or body surface area determines dosing, we selected a dosing quantity based on an average weight or body surface area of a typical patient. Some drugs with dosing ranges that were too variable to calculate an accurate average cost were excluded from analysis.
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