These are the most expensive meds in the US

By Naveed Saleh, MD, MS
Published October 8, 2021

Key Takeaways

The cost of specialty prescription drugs can be staggering. In 2020, the prices of 180 widely used specialty prescription drugs went up 4.8%, according to the results of a study by the AARP Policy Institute. Of note, this rise outpaced general inflation by 3.5 times in 2020.

In other results, the AARP found that the average annual cost of therapy for a single specialty medication used for a chronic condition was greater than $84,000 in 2020. Furthermore, the average annual price of therapy for specialty prescription drugs was nearly 13 times higher than the average annual price of therapy for brand-name prescription drugs.

Recently, GoodRx published a list of the most expensive drugs in the United States. Let’s take a closer look at the top five.

5. Luxturna (voretigene neparvovec-rzyl)

Annual price: $850,000

Luxturna (voretigene neparvovec-rzyl) is an adeno-associated virus (AAV) vector-based gene therapy that is indicated for patients with biallelic RPE65 mutation-associated retinal dystrophy. Of note, retinal dystrophies are degenerative diseases of the retina, which are highly heterogeneous. To use this drug, patients must harbor viable retinal cells, as determined by the specialist.

This biologic is not dispensed by a pharmacy, and necessitates physician administration of one vial into each eye. Although only one vial is injected into each eye during the course of a lifetime, each dose costs a whopping $425,000. There is currently no alternative treatment for this drug.

Like other biologics, this therapy is expensive because of the complexity of the manufacturing process, the use of live materials to create the drug, and all the money invested in research and development, which is ultimately passed to the consumer.

4. Myalept (metreleptin)

Annual cost: $889,904

This leptin analog is utilized along with diet as a replacement therapy to treat complications associated with leptin deficiency in those with congenital/acquired lipodystrophy.

Lipodystrophy is a constellation of rare syndromes resulting in the loss of fat from some parts of the body, and accumulation in other areas. These locations include the liver and other organs. This condition is either congenital or acquired.

Unlike other drugs on this list, this therapy is dispensed at the pharmacy. It costs $74,159 for a month’s supply, with patients self-administering once daily. There are no alternative treatments for lipodystrophy.

3. Danyelza (naxitamab-gqgk)

Annual cost: $977,664

This biologic was approved in November 2020 and is a breakthrough therapy and orphan drug, which was greenlighted by the FDA under accelerated approval. It is a GD2-binding monoclonal antibody that is indicated, along with granulocyte-macrophage colony-stimulating factor (GMCSF), for the treatment of children 1 year of age and older, as well as adult patients, diagnosed with relapsed or refractory high-risk neuroblastoma at the level of the bone or bone marrow. Before the initiation of therapy, patients must have exhibited a partial response, minor response, or stable disease on previous therapy.

This biologic costs $20,368 per vial, with patients needing about 48 vials per year.

2. Zokinvy (lonafarnib)

Annual cost: $1,032,480

This drug is a farnesyltransferase inhibitor and is indicated in patients 12 months of age and older with a minimum body surface area who are at risk of death due to Hutchinson-Gilford Progeria Syndrome. This syndrome causes premature aging, and this drug is the only treatment approved for the condition.

Patients typically take around 200 mg of Zokinvy a day, and the drug costs $717 per 50-mg capsule.

1. Zolgensma (onasemnogene abeparvovec-xioi)

Annual cost: $2,125,000

This biologic has remained the most expensive drug on record since it was first approved in May 2019. It needs to be administered only once.

This therapy is an adeno-associated virus vector-based gene therapy used to treat spinal muscular atrophy in children aged 2 years or fewer. Patients harbor bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

On a final note, because these drugs can cost many multiples of what the average person makes per year, and insurance often falls short of covering the cost, financial aid is available. For instance, Aegerion Pharmaceuticals has an assistance program to help some patients pay for Myalept mentioned above.

Now that you know about the most expensive prescription medications, check out America’s top-prescribed medications on MDLinx.  

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