The Most Expensive Prescription Drugs in the World

The business of developing and selling medications is a huge industry, with companies bringing in billions in revenue every year. Globally, sales of prescription drugs are expected to hit $1.2 trillion in 2024. The most expensive medications are developed to fight rare conditions that are caused by genetic abnormalities.

This list of drugs represents the value of each drug over a month's time. Some of these medicines per dose cost are not as high as others, but the number of doses someone has to take in a month place the drug on the list. Zolgensma is unique among this list as it only requires one dose to be effective. However, at over $2 million for one dose, it is the most expensive.

01
Zolgensma

Priced at a hefty $2.1 million per dose and approved by the Food and Drug Administration in 2019, Zolgensma (by Novartis) was created to treat spinal muscular atrophy (SMA).

This genetic condition causes deterioration in motor neurons that control muscles, generally affecting the body's core muscles. There are four general types of SMA that affect people differently.

Zolgensma addresses all four types of SMA because it is a gene therapy drug designed to replace the defective gene that causes the condition. It is delivered in a liquid form by infusion into a vein, and patients only require one dose.

02
Actimmune

Chronic granulomatous disease is an inherited condition that prevents a body from fighting off infections from certain types of bacteria and fungus.

Patients typically take Actimmune three times a week. The list price for Actimmune is $57,310 per six-milliliter vial. At around 12 vials a month, $687,720 per month is spent on this treatment.

03
Acthar

Acthar is prescribed to treat infantile spasms or seizures. This condition typically affects children between the ages of four to 11 months, but Acthar is approved to treat children up to two years old. It's also used to treat a variety of conditions in adults, including multiple sclerosis.

It costs over $40,000 per 5mL vial. It can only be used at full doses of 75 units, twice daily, for a few weeks before being tapered. Multiple sclerosis requires 80-120 unit doses for two to three weeks.

The cost per dose is about $8,1243, which brings daily costs to about $16,246. For a two week period, approximate costs would be about $227,444. If the drug were to be used for a month it would cost around $457,800.

04
Ravicti

Urea Cycle Disorder affects close to one in 30,000 newborns. This condition causes a buildup of ammonia in the bloodstream which can be fatal within a very short period if not treated.

If taken three times a day as prescribed for a month, with a list price of $5,017 for one bottle of 25ml, the monthly cost would be $421,428.

05
Myalept

Myalept is used to treat generalized lipodystrophy, a condition where the people affected have virtually no fat on their body which affects the body's production of leptin. Leptin helps control certain metabolic processes.

With the necessary doses of 30 injections per month and a list price of $4,846 for the powder, plus the cost of sterile water to mix it with, the monthly price tag is around $145,350.

06
Brineura

Brineura was developed to fight CLN2 disease, which affects the nervous system. The condition usually presents itself between ages two and four. Many of those afflicted make it into their teenage years, but most do not survive beyond adolescence.

A study in 2018 indicated that Brineura was effective in slowing the rate of decline of motor and mental functions.

While this treatment appears to help those affected by the condition, it is costly at $28,198 per kit, while requiring one kit every other week. This brings the monthly total for the treatment to $56,396.

07
Oxervate

Oxervate was developed to treat neurotrophic keratitis, which is loss of vision due to degeneration of the top layer of the cornea caused by damage to the fifth cranial nerve. 

The dosages are prescribed for six times a day at two-hour intervals. You normally receive a weekly carton with seven vials.  One carton is roughly $11,690, bringing a monthly treatment cost to $46,760 (for one eye). 

The treatment is recommended for eight weeks, bringing the total treatment cost to about $93,520 (again, for one eye).

08
Takhzyro

Designed to treat the condition of hereditary angioedema, Takhzyro costs $23,050 for one dose every two weeks initially. Treatments can be slowed to one dose every four weeks, reducing the cost, but only if the patient is responding well.

For the initial monthly dosage, the cost is $46,100.

09
Daraprim

Daraprim treats toxoplasmosis, an infection caused by the Toxoplasma gondii parasite. 

This medication costs about $23,500 for 30 tablets (which is generally enough for a one month supply), given one tablet per day for one to three weeks. A reduction in dosage occurs after three weeks, and patients take it for an additional four to five weeks.

10
Cinryze

Another drug developed to treat hereditary angioedema, this medication prevents the swelling attacks the condition causes.

Used once every three or four days, Cinryze costs about $2,890 per dose. At eight doses per month, roughly $23,120 is spent.