10 of the Most Expensive Medical Treatments and Medications in the U.S.

Photo Courtesy: AnnaStills/iStock

Prescription medications are a vital element of healthcare for many people in the United States. But, like other treatments, their costs can vary widely, from affordable out-of-pocket pricing that becomes even more economical when a patient has health insurance to surprisingly expensive — sometimes surpassing the million-dollar mark in annual costs for a single medication.

While it’s not completely clear how pharmaceutical companies determine pricing for many drugs, some of the most expensive medications and treatments have a few features in common. They tend to be more costly to manufacture, for one, and they’re often specialty treatments like gene therapies or biologic drugs that may be life-saving.

It’s also important to note that there is a difference between the listed price of medications and what price is ultimately paid for them. Pharmaceutical companies will set a price for a drug, but then insurance companies negotiate with pharmacies to determine the price that will be paid for that drug. So the difference between what your insurance company pays for your prescription drugs is different than the retail price listed for that drug, and the cost you pay out of pocket will be different still. If you need to know how much a medication is going to cost for you out of pocket, speak with your insurance company.

You may be able to save money on certain prescription medications by requesting generic versions from your healthcare provider or obtaining different forms of financial assistance. But, ultimately, some treatments and drugs remain much more expensive than others. Here’s a look at 10 of the most expensive medications and treatments in the United States.

Skysona — Around $3 Million

Skysona is a gene therapy medication developed to treat a condition called cerebral adrenoleukodystrophy, or ALD. ALD is an inherited condition that impacts brain function and results in hearing loss and blindness. Skysona is given as a one-time treatment, and the price is set at $3 million. Its high cost arises from the complex, extensive research and development that are involved in producing gene therapy medications.

Zynteglo — Around $2.8 Million

Zynteglo is a one-time medication that was was developed to treat a condition called beta-thalassemia, which is an inherited blood disorder. It limits the amount of hemoglobin a person’s body produces. Hemoglobin is a protein in blood that helps carry oxygen throughout the body. Zynteglo is a gene therapy drug that costs around $2.8 million, which accounts for its high cost. However, because it’s so new, the biopharmaceutical company that developed it will offer refunds if the drug doesn’t work for patients as expected.

Zolgensma — Around $2.1 Million

Zolgensma, another of the most expensive drugs in the U.S., was developed to help treat a condition called spinal muscular atrophy. This inherited neurodegenerative disease typically affects children and can cause difficulty in using their muscles. Zolgensma is a gene therapy medication that’s given as a one-time treatment and is intended for children aged 2 and under. Because it falls within the category of precision medicine, it has a high cost at around $2.1 million, and insurers often need to pay for it in installments.

Danyelza — Around $1.5 Million to $2 Million

Danyelza is a type of treatment for neuroblastoma, a rare form of cancer that affects nerve cells and tissues. This medication is injected, and patients take it every few weeks while undergoing cancer treatment. A vial of Danyelza costs around $23,000, and patients may need around 48 vials annually, which contributes to the overall high cost of treatment with this drug.

Zokinvy — Around $1 Million per year

Zokinvy is the first medication available to treat Hutchinson-Gilford progeria syndrome, a disease that causes premature aging. It works by preventing the buildup of proteins that interrupt cell function and result in early cell aging. Zokinvy dosages are calculated based on a patient’s body size, and it is given as an oral medication daily for up to three years. The price of this medication is over $1 million per year, which was the most expensive drug as of early 2021. 

Folotyn — Around $30,000 per month

Folotyn is an injected medication developed to treat peripheral T-cell lymphoma, a type of cancer in immune system cells, when other treatment options haven’t worked. It is also given to people who have relapsed T-cell lymphoma. The monthly cost of this medication is $30,000, and most patients will need to receive this treatment for a few months.

Actimmune — Around $52,000 per month

Actimmune is part of a regimen of medications that are used to treat a genetic disorder called chronic granulomatous disease, or CGD. People who have CGD are more susceptible to severe infections, and Actimmune gives a patient’s body more of a special type of protein that helps them fight those infections. It costs over $52,000 per month, a high cost that’s driven in part by its rare disease designation. 

Brineura — Around $702,000 per year

Brineura is a prescription medication that treats CLN2 disease, a genetic disorder that affects a patient’s nervous system. CLN2 is a pediatric disorder that causes seizures, loss of motor skills, and intellectual disabilities. Brineura helps to slow the loss of walking and crawling abilities in children with CLN2. This medication is given every other week, and the cost per dose is $27,000, which adds up to a cost of $702,000 per year. CLN2 is a rare disease, which contributes to the high cost of Brineura.

Soliris — Around $650,000 per year

Soliris is a treatment option for several diseases, including paroxysmal nocturnal hemoglobinuria, which causes red blood cells to break down, and myasthenia gravis, which is an autoimmune disease of the nerves and muscles. It’s given as an intravenous solution and binds to specific proteins that lead to the process of red blood cell breakdown. The annual cost of Soliris is listed at over $650,000. Soliris is effective at treating genetic diseases that were once highly fatal, which accounts for the medication’s high cost.

Ravicti — Around $800,000

Ravicti was developed to treat a condition called urea cycle disorder, which causes patients to have high levels of ammonia in their bodies. It works by binding to and removing the ammonia that accumulates in blood and brain tissues. It is a lifelong daily medication that costs just under $800,000

Medical content reviewed by Brittany Stopa, MPH.

Further Reading:

  1. Anderson LA. “10 of the most expensive drugs in the US.” Last updated 17 Oct 2022. https://www.drugs.com/slideshow/top-10-most-expensive-drugs-1274
  2. Congressional Budget Office. “Prescription drugs: spending, use, and prices.” Last updated 21 Jan 2022. https://www.cbo.gov/publication/57772
  3. Patel J, Burger A, McDermott M. “Clinical overview: Skysona for early cerebral adrenoleukodystrophy.” Published 10 Nov 2022. https://www.pharmacytimes.com/view/clinical-overview-skysona-for-early-cerebral-adrenoleukodystrophy
  4. Pagliarulo N. “With $2.8M gene therapy, Bluebird sets new bar for US drug pricing.” Published 18 Aug 2022. https://www.biopharmadive.com/news/bluebird-bio-gene-therapy-price-zynteglo-million/629967/
  5. Cumbers J. “Why some drugs cost $2.1 million per dose and how one company plans to change this.” Published 16 Dec 2021. https://www.forbes.com/sites/johncumbers/2021/12/16/why-some-drugs-cost-21-million-per-dose-and-how-one-company-plans-to-change-this/?sh=39b1ef0a8953
  6. Drugs.com. “Danyelza prices, coupons and patient assistance programs.” Accessed 26 Apr 2023. https://www.drugs.com/price-guide/danyelza
  7. Liu A. “Ultra-rare disease specialists top 2021’s pricey drugs list, but Takeda, Merck KGaA meds rank, too.” Published 09 Mar 2021. https://www.fiercepharma.com/pharma/ultrarare-disease-drug-ascends-to-top-most-expensive-pharmacy-drugs-u-s-2021
  8. Fierce Pharma. “$30K per month: Too much for a cancer drug?” Published 07 Dec 2009. https://www.fiercepharma.com/pharma/30k-per-month-too-much-for-a-cancer-drug 
  9. Christensen J. “The 5 most expensive drugs in the United States.” Published 11 May 2018. https://www.cnn.com/2018/05/11/health/most-expensive-prescription-drugs/index.html
  10. Sagonowsky E. “With FDA nod, BioMarin prices orphan drug Brineura at $702K, but plans hefty discounts.” Published 27 Apr 2017. https://www.fiercepharma.com/pharma/biomarin-picks-up-fda-approval-for-orphan-drug-brineura 
  11. Sagonowsky E. “AstraZeneca’s Soliris needs a 97% to 98% discount in myasthenia gravis, ICER says.” Published 20 Oct 2021. https://www.fiercepharma.com/pharma/astrazeneca-s-soliris-needs-a-97-to-98-discount-myasthenia-gravis-icer-says 
  12. Pharmaceutical Technology. “Top five most expensive drugs in the US.” Published 24 June 2019. https://www.pharmaceutical-technology.com/features/most-expensive-drugs-us/