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10 of the Most Expensive Drugs in the US

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Jan 6, 2022.

Who Can Afford Medications?

Prescription drugs are one of the most important -- if not the most important part -- of a patient's health care regimen. But drugs are expensive, and costs just seem to be rising.

  • A medication can ease a simple headache, as with an affordable and generically available pain reliever like acetaminophen (Tylenol).
  • A drug may be life-saving, say one of the novel biologic immunotherapy checkpoint inhibitors like pembrolizumab (Keytruda) that can target a potentially deadly melanoma skin cancer an dother malignancies.

Either way - a patient should be able to afford or have coverage for the treatments they need to stay alive.

But you have probably found yourself in a situation where the cost of treatment was just too high.

  • Are your annual medical deductibles so out of reach so that it requires you to borrow money to pay your off your bills?
  • Do the bills from the doctor pile up on your table as you try to figure out how pay them?
  • Did you skip filling that prescription last month because you needed to buy groceries for your family instead?

Why are drugs so expensive?

How much you pay for medication is dependent upon many factors, such as:

  • Whether or not you have insurance coverage for drugs.
  • If a drug is paid for on the health insurance formulary.
  • Any rebates or discounts from the manufacturer
  • Your prescription deductibles and copays.
  • If it's a specialty drug, gene therapy, or biologic drug, considered some of the highest priced drugs today.
  • Any financial assistance you might be eligible to receive from either the government (Medicaid, Medicare), discount coupons, or free samples from your doctor.
  • The price set by the manufacturer and their interaction with pharmacy benefit managers (PBMs).
  • Generic or biosimilar availability.

The complicated manufacturing processes involved in some of the most advanced therapies today play a part in high costs. But some expensive drugs are just repurposed generics, too.

How a drug's price is determined is complicated and for most of us remains a true mystery. There is a lack of transparency in how drugs are priced by the pharmaceutical company. The true cost of any drug may be hard to determine until you're at the pharmacy or get a bill from your insurance.

Learn More: FAQs on Generics and Biosimilars

Zolgensma (onasemnogene abeparvovec-xioi)

So what is the world's most expensive single-dose drug? It's Zolgensma (generic name: onasemnogene abeparvovec-xioi), a life-saving gene therapy approved in May 2019 to treat pediatric Spinal Muscular Atrophy (SMA).

Zolgensma's total cost is $2.125 million, according to Novartis, the manufacturer. It's considered the world's most expensive single-dose drug. But over a lifetime, Biogen's SMA drug Spinraza (nusinersen) actually costs much more. Spinraza can cost $4 million over a period of 10 years as it is not given as a one-time drug.

SMA is a rare genetic motor neuron disease that results in problems with breathing, holding up your head, and swallowing. The majority of children with SMA do not survive past early childhood due to an inability to breathe.

Zolgensma is given as a one-time intravenous (IV) dose. It works by replacing the defective or missing survival motor neuron 1 (SMN1) gene, and is considered potentially curative.

Novartis allows insurers to pay for this drug in installments to make it easier to afford, but some insurers may not pay for it. You can check with your doctor and Novartis Gene Therapies to learn more about cost assistance. Call 1-888-NOW-NOVA (1-888-669-6682) Monday - Friday, 8:30am - 5:00pm ET for more cost information.

Danyelza (naxitamab-gqgk)

In Nov. 2020, the FDA approved Danyelza (naxitamab-gqgk) 40 mg/10 mL, used in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of patients one year of age and older with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow. This medicine is given as an infusion into a vein by a healthcare provider.

Neuroblastoma is a solid tumor that occurs in the nervous system outside of the brain in children. Some tumors can be treated, but the majority are aggressive.

The cost for one vial Danyelza intravenous (IV) solution (40 mg/10 mL) is around $22,018. Over one year, the cost for this drug could range between $1.5 to $2 million, although most patients would not take it for this long. Treatment may be stopped due to disease progression or intolerable side effects.

In one study, only 8% of patients continued for longer than one year. Your doctor will decide how long to treat you with this medicine.

Danyelza is also used in combination with a medicine called granulocyte-macrophage colony-stimulating factor (GM-CSF), which adds to the cost of therapy.

For cost assistance information contact Danyelza Y-mAbs Connect Co-pay Program at 833-339-6227 and visit the Price Guide.

Zokinvy (lonafarnib)

Approved in Nov. 2020, Zokinvy (lonafarnib) is used for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies (PL). Zokinvy is indicated in adult and pediatric patients 12 months of age and older with a body surface area (BSA) of 0.39 m2 and above.

Progeria and Progeroid Laminopathies are very rare genetic premature aging diseases that accelerate death in young patients. Untreated children with Progeria die of heart disease at an average age of 14.5 years of age. Roughly 20 children and young adults are known to have the disease in the US.

The cost for Zokinvy is about $22,500 for 30 of the 50 mg capsules and $33,600 for 30 of the 75 mg capsules. Dosing is based on body surface area, but as an example, in a patient taking four 50-mg capsules per day, the cost would be roughly $1.08 million per year.

For cost assistance, contact Eiger OneCare, available Monday through Friday from 9 AM to 5 PM Eastern Time at 1-833-MYEIGER (1-833-693-4437).

Learn More: Zokinvy Prices, Coupons and Patient Assistance Programs

Folotyn (pralatrexate)

Folotyn (pralatrexate), FDA-approved in 2009 from Allos Therapeutics, is used as a last chance cancer medication for patients with relapsed or refractory peripheral T-cell lymphoma (PTCL), an aggressive form of non-Hogkins's lymphoma.

  • PTLC is a relatively rare disease, occurring in less than 9,500 patients each year in the U.S.
  • Folotyn is classified as a cytotoxic antimetabolite (folate antagonist) cancer agent and it's given by intravenous injection. It is given the disease progresses or there in unacceptable side effects.

Folotyn dosing is based on body surface area and is given in cycles, so cost can vary, but an average annual cost can run close to $900,000. However, in studies the median time that patients stayed on treatment was 70 days (with a range between 1 day to 1.5 years).

For financial assistance information, conatct the Folotyn STAR Copay Assistance Program at 888-537-8277.

Actimmune (interferon gamma-1b)

Actimmune (interferon gamma-1b) from Horizon also falls in the very expensive group of genetic disease treatments.

Actimmune is indicated:

  • to treat severe, life-threatening osteopetrosis (the formation of abnormally dense bone, as opposed to osteoporosis, which is brittle bone)
  • to lower the occurrence of serious infections due to chronic granulomatous disease.

Actimmune is given as a subcutaneous (under the skin) injection three times per week. The sticker shock per patient can be palpable, over $819,000 per year, for patients using the 50 mcg/m2 dose.

To learn about cost or copay assistance, conatct the Horizon Co-Pay Assistance Program for Actimmune at 877-305-7704 and review the Actimmune Price Guide.

Brineura (cerliponase alfa)

In April 2017, the FDA approved Brineura (cerliponase alfa), from BioMarin Pharmaceuticals, for a specific form of Batten disease.

  • Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, is a rare inherited disorder that primarily affects the nervous system. It's devastating and patients often require the use of a wheelchair by late childhood. These children typically do not survive past their teens.
  • Batten disease typically begins in early childhood - between the ages of 2 and 4 - with symptoms such as: language delay, seizures and difficulty coordinating movements.
  • Brineura is an enzyme replacement therapy for tripeptidyl peptidase-1 (TPP1). It's the first FDA-approved treatment to slow the decline of walking disability in symptomatic children 3 years of age and older with CLN2.

Brineura runs close to $763,000 per year based on a price of $29,335 per kit. The recommended dosage of Brineura in pediatric patients 3 years of age and older is 300 mg administered once every other week by intraventricular infusion (infusion into the fluid of the brain) through a special port.

An orphan drug for this rare disease, Brineura joins others in the long list of drugs approaching $1 million per year of treatment. Orphan drugs are intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S.

BioMarin Co-pay Assistance Programs are available for Brineura for eligible patients. Contact BioMarin Rare Connections at 1-866-906-6100 Monday-Friday, 6AM-5PM PT for more information.

Soliris (eculizumab)

Soliris (eculizumab), from Alexion Pharmaceuticals is a specialty drug FDA-approved:

  • to treat a rare, chronic blood disease called atypical hemolytic uremic syndrome (aHUS)
  • to prevent the breakdown of red blood cells in people with paroxysmal nocturnal hemoglobinuria (PNH)
  • to treat myasthenia gravis in adults
  • to treat neuromyelitis optica spectrum disorder (NMOSD) in adults.

It is a monoclonal antibody, only available through the Soliris REMS program, and falls within treatments of genetic diseases -- one of the most costly groups of diseases to treat. Soliris used to be considered the most expensive drug in the world, but there are now others that far exceed it.

Soliris is supplied as one 300 mg/30 mL (10 mg/mL) single-dose vial and that costs about $6820 per vial. It's yearly price tag can range from $566,000 to $764,000 per patient, per year, depending upon the condition being treated and body weight (in patients under 18 years of age).

To learn more about drug cost assistance visit the Price Guide for Soliris or call Alexion at 888-765-4747.

Ravicti (glycerol phenylbutyrate)

Urea cycle disorder definitely classifies as an orphan disease as it only affects about 2,100 people in the U.S

Ravicti (glycerol phenylbutyrate), from Horizon Pharma, is a treatment approved to help prevent a build-up of toxic ammonia in the blood in children from birth and older. Ammonia in the blood and tissues and can lead to permanent brain damage, coma, or death.

The cost for Ravicti oral liquid (1.1 g/mL) is around $5,514 for a supply of 25 milliliters. Ravicti is usually taken 3 times per day.

The price tag for Ravicti, given as a liquid taken by mouth, averages about $700,000 per year of treatment. However, these costs will vary based on the specific dose. If you switch from sodium phenylbutyrate to Ravicti, your dose will not be the same. Follow your doctor's dosing instructions very carefully. For questions about Ravicti cost contact a Horizon Therapeutics Patient Access Liasion at 1-855-823-7878.

Lumizyme (alglucosidase alfa)

Lumizyme (alglucosidase alfa) from Genzyme is a lysosomal glycogen-specific enzyme used to treat Pompe disease.

  • In this disease, excess glycogen causes worsening muscle weakness (myopathy) and can affect the heart, skeletal muscles, liver and nervous system.
  • This specific enzyme is needed to break down glycogen, a stored form of sugar used for energy.
  • Lumizyme replaces this enzyme and was approved for the Pompe disease indication in 2014.

While this drug may seem like a bargain at about $905 per vial, each patient, on average, would need about 56 vials per month, for a 70 kg (154 lb) person. Based on this, Lumizyme could have a price tag running about $51,000 per month or close to $612,000 per year.

The manufacturer of Lumizyme offers copay assistance for eligible patients. Call 800-745-4447 to learn more about the Lumizyme Sanofi Genzyme Co-Pay Assistance Program.

Finished: 10 of the Most Expensive Drugs in the US

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