May 19, 2024

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2023 Top 10 Most Expensive Drugs in United States

2023 Top 10 Most Expensive Drugs in United States


2023 Top 10 Most Expensive Drugs in United States.

2023 Top 10 Most Expensive Drugs in United States




Hemgenix – $3.5 million

Hemgenix , an adeno-associated virus vector-based gene therapy developed by CSL Behring and uniQure and approved in November 2022, is the first FDA-approved one-time gene therapy for hemophilia B, priced at one dose $3.5 million.

Hemgenix is ​​indicated for adults with hemophilia B (congenital factor IX deficiency), and delivers the blood clotting factor IX that is deficient in hemophilia B through a one-time intravenous infusion, expresses and produces factor IX protein in the liver, thereby limiting bleeding episodes.


Skysona – $3 million

Skysona is the second gene therapy approved by bluebird bio. Skysona is the first one-time gene therapy approved for the treatment of cerebral adrenoleukodystrophy (CALD), priced at $3 million.

Tom Klima, bluebird’s chief commercial and operations officer, said in the company’s March quarterly report that it was a “data-driven” process that considered “positive clinical outcomes, improvements in quality of life, cost savings for the health system, and patient and family benefits.” potential social impact”.

In addition to the cost of researching and developing gene therapies, manufacturing treatments is expensive and time-consuming. Bluebird says it takes 55 to 60 days to produce a dose of Skysona after cells are collected from a patient.

And the company isn’t going to get huge sales from Skysona. CALD is extremely rare, affecting approximately 50 people each year in the United States, and bluebird expects to treat approximately 10 patients each year.


Zynteglo – $2.8 million

In 2019, Zynteglo was approved by the European Union to treat β-thalassemia, and the price was 1.575 million euros (about 1.8 million U.S. dollars).

It announced its withdrawal from Europe in 2021 because the government was unwilling to give Zynteglo a fair and reasonable price after two years of failed negotiations with the reimbursement authority.

In August 2022, Zynteglo will be approved in the United States, priced at US$2.8 million. The company notes that this is a relative bargain compared to the cost of standard care, which can cost more than $6 million over a patient’s lifetime.

And Zynteglo promises that if the treatment effect is not achieved, 80% of the treatment cost will be refunded.



Zolgensma — $2.25 million

Zolgensma is a one-time AAV gene therapy developed by Novartis for the treatment of spinal muscular atrophy, which was approved in the United States in 2019.
By comparison, Biogen’s approved SMA drug Spinraza costs $750,000 in the first year and $375,000 thereafter. The cumulative cost of Spinraza was higher than Zolgensma from the fifth year onwards, excluding the additional administration fees associated with Spinraza injections.

A Novartis spokesman said Zolgensma’s pricing “reflects the transformative nature and benefits of gene therapy” and “the long-term value it provides”. Novartis already has favorable insurance policies for Zolgensma, covering about 97% of commercially insured patients and 86% of Medicaid patients.

The latest data show that treatment with Zolgensma can be effective for up to 7.5 years after the onset of SMA symptoms . During follow-up, three additional patients managed to stand with assistance.



Myalept – $1.26 million

Myalept, developed by Amryt, is a recombinant human leptin protein analog that was approved for the treatment of Leptin deficiency in 2014. Before the approval of gene therapy, Myalept took the lead in the annual ranking of the world’s most expensive drugs status.
The drug is the only drug approved to treat leptin deficiency, so the price is rising every year. In 2021, Myalept will have annual sales of $141 million.

Currently, Myalept is owned by Chiesi Farmaceutici as Amryt is acquired by Chiesi.


Zokinvy — $1.07 million

Zokinvy is an Ftase inhibitor developed by Merck and approved in 2020 for patients with Hutchinson-Gilford premature aging syndrome. Most of these patients die of heart disease or stroke before the age of 15, with an average annual fee of 107 Ten thousand U.S. dollars.


Danyelza — $1.01 million

Danyelza is a GD2 monoclonal antibody of Y-mAbs. It will be approved in the United States for relapsed or refractory high-risk neuroblastoma in 2020.  It is the only commercial product of Y-mAbs. chemical products.

According to the statistics of GoodRx in 2022, the price of each bottle of medicine exceeds 21,000 US dollars, and each patient spends about 1.01 million US dollars per year.

Y-mAbs stated in its financial report that the drug will generate revenue of US$49.3 million in 2022, an increase of 50% compared to 2021. It is expected that the product’s revenue in 2023 will be between US$60 million and US$65 million.



Kimmtrak – $975,500

Kimmtrak is a TCR-scFv fusion protein developed by Immunocore. The TCR part targets gp100, the scFv part targets CD3 to recruit T cells, and the scFv is fused to the β chain of TCR.

It is the first approved TCR therapy in the world for the treatment of adult patients with HLA-A*02:01 positive unresectable or metastatic uveal melanoma (mUM) .

Kimmtrak is packaged in single-use vials, with a weekly infusion, $18,760 per bottle, and an annual fee of $975,520. In 2022, Kimmtrak sales will be £117 million ($141 million).


Luxturna – $850,000

Luxturna, an AAV gene therapy, is the world’s first gene therapy drug for ophthalmology. It was developed by Spark Therapeutics, a subsidiary of Roche. It was approved in the United States in 2017 for vision loss caused by double-copy RPE65 gene mutations but retaining a sufficient number of viable retinas. Cells for Pediatric and Adult Patients.

The gene therapy uses AAV2 as a carrier to introduce a functional copy of the normal RPE65 gene into the retinal cells of the patient, so that the corresponding cells express the normal RPE65 protein, making up for the RPE65 protein deficiency of the patient, thereby improving the patient’s vision.

Luxturna is a subretinal injection and costs $850,000, or $425,000 per eye.

Folotyn — $842,600

Folotyn is a dihydrofolate reductase inhibitor from Acrotech , which was approved for marketing in the United States in 2009 for the treatment of relapsed or refractory peripheral T-cell lymphoma.

In January 2022, Acrotech raised the price of the drug by 3%, bringing the annual fee to $842,585. Then in December last year, Fresenius Kabi launched a generic drug of Folotyn, which may have a certain impact on Folotyn’s sales.

Folotyn attracted pricing scrutiny immediately after it was approved in 2009. That year, The New York Times highlighted the drug’s then-market price of $30,000 a month, including quoting a UnitedHealth insurer executive who called it “unconscionable.”







2023 Top 10 Most Expensive Drugs in United States

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Important Note: The information provided is for informational purposes only and should not be considered as medical advice.