Medicines closely relate to everyone, and their price is a hot topic around the world.
In the past two years, with the listing of gene therapy, drug price records have been continuously refreshed. Drug companies are putting a high price tag on gene therapies not only because they are expensive to develop and prepare, but also because of the potential for a one-time cure.
In addition, rare diseases are also an area where sky-high drugs frequently appear. Due to the small number of rare patients, small market, and high development costs, pharmaceutical companies are generally not enthusiastic about research and development, and are not willing to invest too much energy in this, so companies want to obtain the same benefits as commonly used drugs, it is necessary to increase the unit price.
On May 22, FiercePharma, a well-known media in the industry, released the report “The Most expensive drugs in the United States in 2023”. This article is arranged here for reference.
NO.1 Hemgenix
Hemgenix, a gene therapy based on adeno-associated virus 5 (AAV5) developed by CSL Behring and uniQure, was approved by the FDA in November 2022 for use in the prophylactic treatment of clotting factor IX or in patients with hemophilia B who have severe or spontaneous bleeding episodes. The product topped the list at $3.5 million per dose.
Despite the high price tag, CSL Behring believes the drug will “deliver significant cost savings to the entire healthcare system” and “significantly reduce the financial burden on patients with hemophilia B by reducing annual bleeding rates, reducing or eliminating preventive treatment and maintaining high factor IX levels for many years.”
NO.2 Skysona
Skysona, a one-time gene therapy using Lenti-D lentiviral vector (LVV) for in vitro transduction, developed by Bluebird Biology, was approved by the FDA in September 2022 to slow the progression of neurological dysfunction in boys aged 4-17 years with early active cerebral adrenoleukodystrophy (CALD).
It is Bluebird’s second gene therapy to hit the market and is priced at $3 million per dose. Bluebird Bio said that in addition to the research and development costs, the preparation of Skysona also requires a lot of manpower, resources and time. For example, it takes 55 to 60 days to produce a dose of Skysona after collecting cells from a patient.
NO.3 Zynteglo
Zynteglo, also a gene therapy developed by Bluebird Bio, was approved by the FDA in August 2022 for the treatment of beta-thalassemia patients of all genotypes in adults, adolescents and children who require regular blood transfusions.
The product is the first one-time treatment approved in the United States to target the underlying genetic cause of beta-thalassemia in patients, offering patients an alternative to conventional red blood cell infusion and iron chelating therapy at a price of $2.8 million per dose. In addition, Bluebird Bio promises to refund 80 percent of the cost of treatment if patients are unable to get out of transfusions.
In fact, Zynteglo was already listed in the European Union in May 2019, priced at 1.575 million euros (about $1.8 million). But because the price was too high and did not meet the cost-effectiveness criteria of national payers, Bluebird Bio announced in August 2021 that Zynteglo would be withdrawn from the European market.
NO.4 Zolgensma
Zolgensma is a one-time AAV9-based gene therapy developed by Novartis and approved by the FDA in May 2019 for the treatment of spinal muscular atrophy (SMA). The product topped the list for years until 2022 and is currently priced at $2.25 million per dose.
Novartis said Zolgensma’s pricing reflects “the transformative nature of gene therapy” and “the long-term value it provides,” with long-term benefits despite the high price tag. By comparison, another SMA drug, Spinraza, costs $750,000 in the first year of treatment and $375,000 annually thereafter.
NO.5 Myalept
Myalept is a recombinant human leptin protein analogue that was approved by the FDA in February 2014 for the treatment of systemic lipodystrophy and leptin deficiency in patients with systemic fat distribution abnormalities. This product is the only drug in the market with the same target, and even there are no other drugs in the late clinical stage of the disease, and patients have few treatment options.
For years, Myalept has led the list of most expensive drugs, currently costing patients up to $1.26 million per year to treat. According to Chiesi, a bottle of Myalept costs $5,867.52, and patients use an average of 18 bottles per month. In early 2021, the price of the product just increased from $5,093 to $5,297 per bottle.
NO.6 Zokinvy
Zokinvy is an FTase inhibitor developed by Eiger Biopharmaceutical for Hutchinson-Gilford progeria syndrome (HGPS or progeria) and progery-like laminosis, approved by the FDA in November 2020. Both indications are ultra-rare, hereditary diseases of premature aging that accelerate mortality in younger patients.
The product also costs more than $1 million a year. Although the dosage depends on the body’s surface area, patients typically take about 200mg of Zokinvy per day. At $746 for a 50mg capsule, the monthly cost would be $89,500. As a result, Zokinvy ranks among the world’s most expensive drugs, with an annual cost of $1.07 million.
NO.7 Danyelza
Danyelza is a GD2 monoclonal antibody developed by Y-mAbs Therapeutics and approved by the FDA in November 2020. Combined with granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of recurrent or refractory high-risk neuroblastoma patients 1 year of age and older with bone or myelopathy who have shown partial response, mild response, or stable disease on prior treatment. In December 2022, the drug entered the Chinese market.
According to GoodRx, in December 2021, the price of Danyelza was increased by 3.5%. Currently, each bottle is priced at more than $21,000, costing patients about $1.01 million a year. Y-mAbs reported sales of $49.3 million for Danyelza in 2022, up 50% year over year.
NO.8 Kimmtrak
Kimmtrak is a T-cell receptor (TCR) therapy developed by Immunocore and approved by the FDA in January 2022 for the treatment of HLA-A* 02:01-positive adult metastatic uveal melanoma.
The product costs $18,760 per bottle, and patients need weekly infusions. This means that patients would have to spend $975,520 per year, but most patients would not be able to receive medication for that long. The Phase III study showed that only 31% of patients were still alive at six months with no disease progression.
NO.9 Luxturna
Luxturna is a gene therapy that was approved by the FDA in December 2017 for the treatment of inherited retinal dystrophy. The product is also the first AAV gene therapy approved in the United States, using AAV2 to deliver the normal RPE65 gene into retinal pigment epithelium (RPE) cells, thereby compensating for the RPE65 gene mutation.
Luxturna, which can only be used in the hospital and requires a doctor to inject the drug into each eye, comes with an expensive price tag of $425,000 per bottle. Spark Therapeutics, however, agreed not to charge the full cost if the drug doesn’t work.
NO.10 Folotyn
Folotyn, a dihydrofolate reductase (DHFR) inhibitor, was first approved by the FDA in 2009 for the treatment of relapsed or refractory peripheral T-cell lymphoma at an annual cost of more than $840,000. In 2020, the product was approved in China.
According to GoodRx, patients typically need to use about 135 vials of Folotyn per year. Without the use of discounts and offers, Folotyn costs more than $6,200 per bottle. As a result, Folotyn still holds its place on the list of the world’s most expensive drugs, even though gene therapies and other innovative products are entering the market.
