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.