Pharmacy Fact: What is Currently the Most Expensive Drug in the United States?

Drug prices in the United States are high and rising,¹ but some medications are truly in a class of their own. When trying determine which drug can be called the most expensive on the market, there are 2 potential answers depending on how you define the question.^2,3

In terms of drugs that can be filled by your local pharmacist, the most expensive medication available—by a considerable margin—is lonafarnib (Zokinvy, Eiger Biopharmaceuticals). Dosage is dependent on body surface area, but most patients take 200 mg a day, which at $717 per 50 mg capsule adds up to $86,040 a month,² or more than $1 million per year.³

Lonafarnib is an orphan drug designed to reduce the risk of death for patients with Hutchinson-Gilford progeria syndrome, a rare genetic disease that results in premature aging. There is currently no cure for this condition, and lonafarnib is the only drug approved to be used in its treatment.²

Lonafarnib isn’t the most expensive drug on the market, however. Including medications that have to be administered by a health care practitioner, one stands out as the most expensive, at more than double the price of Lonafarnib: Onasemnogene abeparvovec (Zolgensma, Novartis). At more than $2.1 million dollars for an annual supply, Zolgensma has been the most expensive drug on the market since its FDA approval in May 2019.³

Zolgensma is approved to treat spinal muscular atrophy (SMA), a rare childhood disorder that results in muscular erosion. This can lead to lung infections and muscle weakness,³ and in its most serious form, it can be fatal before patients reach 2 years of age.⁴ Zolgensma is considered to be a 1-time curative therapy, with many children treated with the drug showing no signs of the disease after treatment.³

Complicating matters even further, Zolgensma may not always be covered by insurance,⁴ leaving parents of patients with SMA to foot the bill themselves in large annual installments.³ However, it’s been argued that the price of Zolgensma is worth paying for what it ultimately offers to patients: the drug is the only 1-time treatment for SMA available. The next best alternative, nusinersen (Spinraza, Biogen), must be administered on a regular basis, with a cost over a 10-year period of more than $4 million.⁵

REFERENCES

• Waxman H, Corr B, Martin K, Duong S. Getting to the root of high prescription drug prices: drivers and potential solutions. Issue Brief (Commonw Fund), 2017, 1-10. Accessed August 12, 2021. https://www.commonwealthfund.org/sites/default/files/documents/___media_files_publications_fund_report_2017_jul_waxman_high_drug_prices_drivers_solutions_report_la_en.pdf
• The 20 most expensive prescription drugs in the U.S.A. GoodRx; February 8, 2021. Accessed August 12, 2021. https://www.goodrx.com/blog/20-most-expensive-drugs-in-the-usa/
• The 10 most expensive drugs in the U.S., period. GoodRx; March 8, 2021. Accessed August 12, 2021. https://www.goodrx.com/blog/most-expensive-drugs-period/
• Zolgensma set a new drug pricing bar. Insurers show some signs of pushback. Biopharma Dive; July 3, 2019. Accessed August 12, 2021. https://www.biopharmadive.com/news/zolgensma-set-a-new-drug-pricing-bar-insurers-show-some-signs-of-pushback/558101/
• Cohen, J. At over $2 million Zolgensma is the world’s most expensive therapy, yet relatively cost-effective. Forbes of, 5. Accessed August 12, 2021. https://almashines.s3.dualstack.ap-southeast-1.amazonaws.com/assets/media/files/488_1616563699_bc12f43af51c4ba03004486fbd5f219f.pdf