The most expensive drugs in the world — starting at the low price of $1 million
The pursuit of treatments for rare diseases has come with a steep cost: The average price of new drugs increased 35% in the U.S. in 2023, according to an analysis by Reuters.
That price hike comes as the pharmaceutical industry is embracing treatments for what are known as orphan diseases, or illnesses that typically affect a small population, usually 200,000 people or less. Arriving in a market with a lack of alternative treatment options, drug companies can — and do — price their treatments at sky-high rates.
Here are 10 of the world’s most expensive drugs — based on list price and not the actual price patients pay after any manufacturer rebates.
Immunocore’s Kimmtrak — $1.1 million
Kimmtrak was approved by the U.S. Food and Drug Administration (FDA) in 2022 to treat an eye cancer known as metastatic uveal melanoma (mUM). It costs about $21,434 per vial — which accounts for just a weekly dose. Over a year, that would translate to nearly $1.1 million. About 1,700 Americans are diagnosed with uveal melanoma annually, according to the U.S. National Cancer Institute.
Eiger BioPharmaceuticals’s Zokinvy — $1.2 million
Zokinvy is used to treat Hutchinson-Gilford progeria syndrome, a genetic disease that causes rapid aging. It costs nearly $40,000 for (30) 50mg pills. Depending on the prescribed dosing, that could add up to $1.2 million annually. At any given time there are about 400 children around the world who are living with Hutchinson-Gilford progeria syndrome.
Y-mAbs Therapeutics’s Danyelza — $1.2 million
Danyelza treats neuroblastoma, a rare childhood cancer, found in a patient’s bone or bone marrow. The drug costs $24,323 a vial. Typically patients need 48 vials a year, adding up to nearly $1.2 million. About 600 to 1,000 people are diagnosed with with neuroblastoma every year in the United States.
Amryt Pharmaceuticals’s Myalept — $1.4 million
Myalept treats a leptin deficiency in people with lipodystrophy. The medication about $6,500 per vial. On average patients take about 18 vials a month, resulting in an average annual cost of $1.4 million. There are only about 250 reported cases of lipodystrophy in medical literature.
Novartis’s Zolgensma — $2.1 million
Zolgensma is a one-time gene therapy treatment that for spinal muscular atrophy in children aged two and younger. It was once the most expensive drug in the world, costing $2.1 million.
Bluebird bio’s Zynteglo — $2.8 million
Zynteglo is another one-time, single dose gene therapy. Its made from a patient’s stem cells and is used to treat a rare genetic blood disorder, beta thalassemia. Patients with this condition often require regular blood transfusions. The medication costs $2.8 million.
Bluebird bio’s Skysona — $3 million
Skysona was approved by the FDA in 2022 to treat boys 4 to 17 years old with early, active cerebral adrenoleukodystrophy (CALD), a neurological disorder that could result in the loss of all voluntary movement. The one-time gene therapy costs $3 million. Bluebird estimates that are about 40 new cases of CALD in the U.S. each year.
Sarepta Therapeutics’s Elevidys — $3.2 million
Elevidys is a gene therapy for Duchenne muscular dystrophy (DMD). The muscle disorder, which leads muscle weakness and loss, affects nearly 15,000 boys in the U.S. and 300,000 worldwide, most of whom don’t survive past their late 20s. The one-time therapy costs $3.2 million.
CSL Behring’s Hemgenix — $3.5 million
The previous title holder for world’s most expensive drug was CSL Behring’s Hemgenix. The gene therapy launched in 2022 and is used to treat the bleeding disorder hemophilia B. There are between 30,000 to 33,000 people living with hemophilia in the U.S.
Orchard Therapeutics’s Lenmeldy — $4.25 million
Lenmeldy is a one-time, single-dose infusion made of a patient’s own blood and stem cells to treat infants with early metachromatic leukodystrophy (MLD). The rare genetic disease results in the build up of fatty substances called sulfatides in the cells of the brain, nervous system, and other organs. It can result in loss of motor and cognitive function, and even death. The FDA said that MLD affects one in 40,000 Americans. Last week, Lenmeldy maker Orchard Therapeutics set the price for the treatment at a staggering $4.25 million.