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.
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