9 Most Expensive Pharmaceutical Drugs in the US

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The United States is the world’s leading pharmaceutical market and the sole producer of many expensive, valuable drugs. This makes it more important than ever to know what is the most expensive drug in America because drug spending is something that will continue to rise as doctors prescribe more drugs for their patients.

These drugs have been created through years of research and development to help people who suffer from various ailments or have conditions that could become illnesses if left untreated. Some of these drugs are approved for prescription, while some are only available through an over-the-counter treatment.

Read on and take a closer look at the nine most expensive drugs available in the US.

9. Brineura

Price: $763,000 per year
Manufacturer: BioMarin Pharmaceutical Inc.
Use: To treat neuronal ceroid lipofuscinosis type 2 (CLN2 disease)
FDA Approved Date: April 27, 2017

Brineuraphoto source: Batten Disease News

Treatment for a particular kind of Batten disease known as late-infantile neuronal ceroid lipofuscinosis type 2 is provided with the first-in-class drug Brineura (CLN2). In particular, Brineura aids in the treatment of children patients 3 years of age and older who gradually lose their ability to walk.

The advertised price of Brineura for a package of two 150 mg vials is currently $29,073 after a 3.5% price rise in January. When taking the suggested dosage of 300 mg every two weeks, the annual cost comes to $755,898.

Did You Know?

Through their copay assistance program, manufacturer BioMarin does offer financial aid to insured and cash-paying patients.


8. Actimmune

Price: $819,000 per year
Manufacturer: Horizon Pharma
Use: To treat chronic granulomatous disease (CGD)
FDA Approved Date: Around 1990

Actimmunephoto source: Extreme Living

For the treatment of Chronic Granulomatous Disease (CGD), a pharmacological regimen, Actimmune is typically used. A genetic condition known as CGD impairs certain immune system cells and the body’s capacity to properly fight infections.

It is often identified in childhood. Antibiotics, antifungals, and Actimmune are frequently used in the treatment of CGD, albeit it is not a cure. The progression of severe, malignant osteopetrosis can also be prevented with it (SMO).

Did You Know?

Typically discovered within the first few months of life, SMO is a hereditary condition that interferes with normal bone development.


7. Luxturna

Price: $850,000 per year
Manufacturer: Spark Therapeutics, Inc.
Use: To treat biallelic RPE65 mutation-associated retinal dystrophy
FDA Approved Date: December 19, 2017

Luxturnaphoto source: Arizona Retina Project (University of Arizona)

The drug Luxturna is used to treat adults and kids who have lost their eyesight as a result of hereditary retinal dystrophy, a rare genetic condition affecting the retina (the light-sensitive membrane at the back of the eye). When the condition is brought on by mutations (changes) in the RPE65 gene and individuals still have enough functional retinal cells, Luxturna may be administered.

An enzyme termed all-trans retinyl isomerase, which is essential for retinal cells to function normally, is produced by RPE65.

Luxturna is a category of advanced treatment medication referred to as a “gene therapy product” and includes the active ingredient voretigene neparvovec.

Did You Know?

The said delivering genes into the body is how this kind of medication functions.


6. Folotyn

Price: $900,000 per year
Manufacturer: Allos Therapeutics, Inc.
Use: To treat T-cell lymphoma
FDA Approved Date: September 25, 2009

Folotynphoto source: cancermedicinesnetwork.com

For the treatment of T-cell lymphoma that has spread throughout the body, the antineoplastic (anti-cancer) medication Folotyn (pralatrexate injection) is utilized. When previous treatments have failed to control the patient’s T-cell lymphoma, or if the disease has relapsed, Folotyn is prescribed.

The blood cells that aid in blood clotting and infection defense in your body can be reduced by Folotyn.

A more straightforward bleed or infection might occur. If you see unusual bleeding, bruising, or other infection-related symptoms, call your doctor right once (fever, chills, body aches). If you are expecting, you should avoid using it. The unborn child might be harmed. If you become pregnant while receiving therapy, use an effective form of birth control and let your doctor know.

Did You Know?

While receiving Folotyn treatment, you shouldn’t breastfeed your infant.


5. Myalept

Price: $929,951 per year
Manufacturer: Amryt Pharmaceuticals
Use: To treat leptin deficiency
FDA Approved Date: February 24, 2014

Myaleptphoto source: medicaltourisrael.com

The sole medication on this list that is exclusively available via a pharmacy is Myalept. Leptin deficiency, which results in aberrant fat distribution in the body, is treated with it in individuals with widespread lipodystrophy. The monthly supply price of Myalept is $77,496.

Myalept costs $929,951 a year when self-administered, with the average user using 14 vials per month. There are no more affordable options because Myalept is the only medication that can address this uncommon illness. A few people can get financial aid from Myalept’s maker, Amryt Pharmaceuticals, with the help of these initiatives.

Did You Know?

When compared to the month of September, the price of Myalept is up 4.5% as of January 2022.


4. Kimmtrak

Price: $975,520 per year
Manufacturer: Immunocore Ltd.
Use: To treat adults with a type of eye cancer called ‘uveal melanoma’
FDA Approved Date: January 25, 2022

Kimmtrakphoto source: drugs.com

Adults with “uveal melanoma,” an eye malignancy, can be treated with Kimmtrak. When uveal melanoma cannot be surgically removed or has migrated to other regions of the body, it is employed. Since uveal melanoma is an uncommon condition, Kimmtrak was named an “orphan medicine” on February 19, 2021.

Kimmtrak, the most recent medication to be included on the list, was given FDA approval in January 2022. A healthcare professional administers it by IV infusion, and the usual user uses one vial on a weekly basis as a maintenance dose.

Did You Know?

More than 3 in 10 people may experience the most frequent side effects of Kimmtrak, which include cytokine release syndrome, rash, fever, itching, fatigue, nausea, chills, abdominal (belly) pain, edema (swelling), hypo/hyperpigmentation (change in skin color), hypotension (low blood pressure), dry skin, headache, and vomiting.


3. Danyelza

Price: $1,011,882 per year
Manufacturer: Y-mAbs Therapeutics
Use: To treat high-risk neuroblastoma
FDA Approved Date: January 27, 2021

Danyelzaphoto source: everyone.org

One particular form of cancer is treated using this drug (neuroblastoma in bone or bone marrow). Monoclonal antibodies, which include naxitamab, are a group of medications. It inhibits or stops the proliferation of cancer cells, which is how it functions.

On the first, third, and fifth days of each treatment cycle, this drug is injected into a vein as prescribed by your doctor. Every 4 or 8 weeks, a treatment cycle typically lasts. Usually, over the course of 30 to 60 minutes, the injection is administered by a medical expert.

Did You Know?

In order to assist reduce the chance of side effects like discomfort, infusion reactions, nausea, and vomiting, doctors typically prescribe additional drugs for you to take both before and during treatment.


2. Zokinvy

Price: $1,073,760 per year
Manufacturer: Merck
Use: Treat Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies
FDA Approved Date: November 20, 2020

Zokinvyphoto source: MPR

In November 2020, the FDA authorized the orphan medicine Zokinvy. Over $1 million a year is a surprising amount to pay for it. For the uncommon genetic condition Hutchinson-Gilford progeria syndrome, which causes accelerated aging, it is the first and only therapy that has been licensed.

People commonly take 200 mg of Zokinvy per day, depending on body surface area; at $746 for every 50 mg capsule, this works up to $89,480 per month. This price reflects an increase of 4% that took place in December 2021.

Did You Know?

Although this medication does not have a copay card, the Eiger OneCare program can aid with insurance coverage and financial assistance alternatives.


1. Zolgensma

Price: $2,125,000
Manufacturer: Novartis Gene Therapies, Inc.
Use: To spinal muscular atrophy
FDA Approved Date: June 19, 2015

Zolgensmaphoto source: Pharmaceutical Technology

For a price tag of over $2 million, Zolgensma is considered the most expensive drug you can get in the US. Spinal muscular atrophy, a significant nerve disorder that results in muscle atrophy and weakness, is a disease that can be treated using the gene therapy drug Zolgensma.

Utilizing this medication has the potential to produce acute significant liver damage or acute liver failure as well as a rise in liver enzyme levels. Before and after receiving a Zolgensma infusion, patients will get an oral corticosteroid, and frequent blood tests will be performed to check on their liver health.

Did You Know?

The AAV9 virus’s innocuous components are used to make Zolgensma, which has been “gutted” to create something akin to an empty envelope into which anything can be introduced.

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