As more rare disease therapies gain approval in recent years, the healthcare industry has increasingly adopted pricey treatments for rare genetic conditions like muscular dystrophies and blood disorders.
This new wave of gene therapies now constitutes some of the world’s most expensive drugs, mainly because of their curative potential and single administration requirement. Their high price tags are also partly attributed to high manufacturing costs, the use of advanced technologies and relatively small target populations.
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By GlobalDataIn contrast, some biologics prove expensive because of ongoing treatment costs. Nonetheless, even if gene therapies are seen as cost-effective, pharmaceutical companies in the US continue to face scrutiny over exorbitant prices.
In this article, Pharmaceutical Technology explores the top ten most expensive drugs in the US, using publicly available data and GlobalData’s Pharma Intelligence Center.
GlobalData is the parent company of Pharmaceutical Technology.
1. Lenmeldy- $4.25m
Lenmeldy (atidarsagene autotemcel) is the most expensive drug on the market with a steep price tag of $4.25m. The gene therapy is developed for children with metachromatic leukodystrophy (MLD), a potentially fatal disorder caused by a mutation in the arylsulfatase-A (ARSA) gene, which leads to the buildup of fats. This causes the breakdown of the myelin sheath—the protective fatty layer surrounding the nerves—leading to a progressive decline in cognitive and motor functions.
Lenmeldy was developed by Orchard Therapeutics, which was acquired by Kyowa Kirin, a Japan-based global specialty pharmaceutical company, for an equity value of $477.6m (JPY70.47bn) in early 2024.
Lenmeldy received a US Food and Drug Administration (FDA) approval for certain patient groups in March 2024. The treatment is a single-administration, personalised infusion. Apart from its high price tag, one of the greatest challenges in the therapy’s uptake will be patient identification if wide-scale post-birth screening is not implemented for early diagnosis.
2. Beqvez and Hemgenix- $3.5m
Two gene therapies for haemophilia B—Beqvez (fidanacogene elaparvovec-dzkt) and Hemgenix (etranacogene)—are tied for second place on this list, both costing $3.5m. Haemophilia B is a rare genetic disorder characterised by inadequate blood clotting due to a deficiency in factor IX, which results in prolonged and more frequent bleeding episodes.
Beqvez’s (fidanacogene elaparvovec-dzkt) April FDA approval marked a critical moment for Pfizer, as it became the company’s first FDA-approved gene therapy. This approval came almost two years after CSL Behring’s Hemgenix was approved in the same indicationa.
Hemgenix—an adeno-associated virus vector (AAV)-based gene therapy—marked the first of its kind treatment for adults with haemophilia B. The therapy temporarily held the title of the “most expensive drug in the world” with its listed $3.5m price, following its approval in November 2022.
In general, haemophilia gene therapies have been scrutinised for the cost and the value they provide to patients and society especially when it comes to transitioning from traditional treatments to new gene therapies.
According to reports by the Institute for Clinical and Economic Review (ICER), haemophilia gene therapies like Hemgenix have been rendered cost-effective after evaluating their clinical efficacy and value compared to factor IX and VIII prophylaxis. However, the long-term efficacy and duration of this “cure” as well as the safety concerns surrounding those therapies remain important uncertainties.
3. Elevidys- $3.2m
The third priciest therapy in the US is Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl) with a price tag reaching $3.2m. In June 2023, Elevidys received accelerated FDA approval, becoming the first gene therapy for treating children aged four to five years with Duchenne muscular dystrophy (DMD) who possess a confirmed mutation in the DMD gene.
A year later, the FDA expanded Elevidys's label to include DMD patients aged four and older with a confirmed mutation in the DMD gene. As part of the post-marketing validation requirements, the Phase III ENVISION study (NCT05881408) is underway to assess Elevidys in non-ambulatory and older ambulatory DMD patients.
Elevidys is a one-time therapy that utilises a recombinant adeno-associated virus (AAV) vector serotype 74 to introduce a micro-dystrophin transgene that encodes Elevidys micro-dystrophin, which contains certain parts of the dystrophin protein. DMD is the most common type of muscular dystrophy in children, predominantly affecting males, caused by mutations in the dystrophin gene.
4. Lyfgenia- $3.1m
Ranked fourth on the list of the costliest therapies is Lyfgenia (lovotibeglogene autotemcel), a cell-based gene therapy developed for sickle cell disease. Sickle cell disease is a genetic, inherited red blood cell disorder caused by a mutation in the beta-globin gene. This mutation alters the shape of red blood cells from the normal disc to a sickle shape, leading to anaemia and vaso-occlusive events.
Lyfgenia, a one-time treatment, utilises a lentiviral vector to incorporate functional copies of the beta-globin gene into the patient’s cells, encouraging the production of anti-sickling haemoglobin, which may help reduce or prevent vaso-occlusive events. Lyfgenia was one of the first gene therapies to secure an FDA approval for sickle cell disease.
The therapy is developed by bluebird bio, which owns three therapies featured on this list.
In the beginning of 2024, the Centers for Medicare and Medicaid Services (CMS) announced it would act as a proxy for negotiations for outcome-based agreements, and pricing rebates for cell and gene therapies, with a focus on sickle disease gene therapies such as Lyfgenia.
5. Skysona- $3m
Skysona (elivaldogene autotemcel) holds the fifth place in the ranking of the priciest US drugs with a staggering $3m price tag. Skysona, also developed by bluebird bio, was the first to slow down the progression of neurologic brain dysfunction in boys aged four to 17 with early, active cerebral adrenoleukodystrophy (CALD).
CALD is a rare, progressive, and fatal neurodegenerative disease that leads to severe and irreversible cognitive, mental, and motor decline. CALD happens due to a mutation in the ABCD1 gene that leads to adrenoleukodystrophy protein (ALDP) enzyme deficiency and prevents fatty acid breakdown, which destroys nerve myelin sheath.
Back in 2022, Skysona was granted an accelerated approval and the company won a rare paediatric priority review voucher following its approval.
Although it possesses curative potential, the one-time therapy is linked to side effects such as haematologic malignancies, including life-threatening cases of myelodysplastic syndrome.
6. Roctavian- $2.9m
BioMarin’s Roctavian (valoctocogene roxaparvovec) is another expensive haemophilia gene therapy that received FDA approval in June 2023. The regulatory success came after a four-year process involving delays and previous rejections and the company set a striking price of $2.9m for it.
Roctavian became the first and only one-time therapy for adults with severe haemophilia A. The therapy has a recombinant AAV5 viral vector carrying a gene that stimulates the production of factor VIII.
Despite its $2.9m price tag, Roctavian's one-time administration holds the potential to significantly enhance a patient’s quality of life. However, similar to CSL’s Hemgenix - the pricing of Roctavian also sparked debate, with ICER initially recommending a fair price of approximately $1.9m.
7. Rethymic- $2.81m
Enzyvant’s Rethymic (allogeneic processed thymus tissue) is the first and only FDA-approved treatment designed to restore immune function in children with congenital athymia, a condition where patients are born without a thymus, leading to severe immunodeficiency and immune system disorders.
With a listed price of $2.81m, this tissue-based therapy is a bioengineered human thymus tissue designed to restore the missing thymic function without the need for donor-recipient matching.
8. Zynteglo- $2.8m
Another gene therapy produced by bluebird bio is Zynteglo (betibeglogene autotemcel or beti-cel), a lentiviral vector gene therapy for treating adults and children with beta-thalassaemia who require regular red blood cell (RBC) transfusions.
In August 2022, the FDA granted Zynteglo an FDA approval, which was priced at $2.8m per dose and momentarily became the most expensive therapy in the world. Bluebird secured another approval for Skysona four weeks later and set an even higher price for it surpassing Zynteglo.
Similar to previously mentioned therapies, Zynteglo uses a patient’s stem cells and is administered as a one-time, single-dose intravenous infusion.
Although Zynteglo could defend its high price tag when compared to the cumulative price of regular blood transfusions over a patient’s lifetime, its cost still drew negative attention. ICER, which assessed its cost-effectiveness, supported bluebird’s pricing and valued Zynteglo at $2.77m per dose, noting that the company pledged to refund 80% of the treatment cost if patients do not achieve transfusion independence.
9. Zolgensma- $2.32m
Novartis’s breakthrough therapy Zolgensma (onasemnogene abeparvovec) is listed for $2.32m. In May 2019, Novartis subsidiary AveXis gained FDA approval for Zolgensma, which is designed to treat children under two years of age with spinal muscular atrophy (SMA) caused by mutations in the SMN1 gene.
SMA is a rare genetic disorder that affects motor neuron function, leading to severe muscle weakness and in its most severe form can cause paralysis or death by the age of two. Zolgensma uses an AAV vector, which targets the genetic root cause of SMA by delivering a functional copy of the SMN gene and halting disease progression.
Although the therapy was pioneering, Novartis faced criticism for submitting manipulated data in its FDA application following Zolgensma’s approval. Safety issues regarding Zolgensma also emerged as the FDA placed a partial hold on the company’s Phase I/II STRONG study due to safety concerns in an animal study.
In August 2022, Novartis announced its intention to update the drug's labeling to indicate that fatal acute liver failure had been reported in connection with its use after two patient deaths were attributed to acute liver failure.
10. Casgevy- $2.2m
The last therapy on this list is Vertex Pharmaceuticals and CRISPR Therapeutics’ first-of-its-kind Casgevy (exagamglogene autotemcel), with a listed price of $2.2m.
In November 2023, Casgevy’s conditional UK approval became a pivotal moment, marking the world’s first CRISPR-based gene therapy approved for sickle cell anaemia and transfusion-dependent beta-thalassemia
Casgevy consists of a genetically modified population of autologous CD34+ cells that have been edited using the pioneering clustered regularly interspaced short palindromic repeats (CRISPR) technology. The therapy is also a one-time single-dose infusion. These engineered human hematopoietic stem and progenitor cells produce elevated levels of fetal hemoglobin (HbF), which can eliminate the need for transfusions in thalassemia patients, and also prevent vaso-occlusive events in sickle cell disease patients.
The groundbreaking therapy was later approved in the US in early 2024.
