8 of the most expensive drugs in biopharma: Unraveling the costliest medications By Willow Shah-Neville 14 minutesmins June 22, 2026 14 minutesmins Share WhatsApp Twitter Linkedin Email Photo credits: freestocks (Unsplash) Newsletter Signup - Under Article / In Page"*" indicates required fieldsInstagramThis field is for validation purposes and should be left unchanged.Subscribe to our newsletter to get the latest biotech news!By clicking this I agree to receive Labiotech's newsletter and understand that my personal data will be processed according to the Privacy Policy.*Company name*Job title*Business email* In recent years, the price of drugs has risen. According to an analysis by Reuters, the average price of new drugs increased by 35% in the U.S. in 2023 year on year, reflecting in part the industry’s embrace of expensive therapies for rare diseases like muscular dystrophy. Gene therapies have emerged as some of the costliest treatments around, largely due to their curative potential and the fact that they only need to be administered once. In this article, we look at eight of the most expensive drugs in biopharma based on U.S. list prices. Table of contentsLenmeldy Cost: $4.25 million Company: Orchard Therapeutics After being given the green light in 2024, Lenmeldy became the first U.S. Food and Drug Administration (FDA)-approved gene therapy for the treatment of children with metachromatic leukodystrophy (MLD), a rare and potentially fatal genetic disease affecting the brain and nervous system caused by a deficiency of an enzyme called arylsulfatase A (ARSA). This leads to a buildup of sulfatides (fatty substances), which destroy the protective layer of the nerves called the myelin sheath. Patients eventually lose the ability to move, talk, swallow, eat, and see. It is estimated that MLD affects one in every 40,000 individuals in the U.S. Lenmedly is a one-time treatment made from the patient’s own hematopoietic stem cells (HSCs), which have been genetically modified to include functional copies of the ARSA gene. Once the stem cells have been collected and modified ex-vivo, they are then infused back into the patient, where they engraft (attach and multiply) within the bone marrow, eventually migrating across the blood-brain barrier into the central nervous system and expressing the functional enzyme. When Orchard Therapeutics announced in March 2024 that Lenmeldy would cost $4.25 million, it officially became the world’s most expensive drug and continues to be so to this day. The company said that the price reflects the value the therapy could deliver to eligible patients and their families, as well as the potential long-term impact the treatment may have on overall healthcare utilization, minimization of productivity loss for caregivers, and life opportunities for patients. Kebilidi Cost: $3.95 million Company: PTC Therapeutics After being approved in 2024, Kebilidi is the second most expensive drug around. It was developed by PTC Therapeutics and became the first gene therapy for treating aromatic L-amino acid decarboxylase (AADC) deficiency, an ultra-rare inherited neurological disorder caused by mutations in the DDC gene. The condition prevents the body from producing sufficient amounts of key neurotransmitters, including dopamine and serotonin, which are essential for controlling movement, behavior, mood, and autonomic functions. As a result, affected children often experience severe developmental delays, low muscle tone, difficulty feeding, involuntary movement disorders, and life-threatening complications, with many never achieving basic milestones such as sitting, standing, or speaking independently. Suggested Articles Eight in vivo CAR T biotechs to follow closely 10 gene therapy companies you should know about Eight areas where gene therapy shines 5 cell therapy companies you should know about The future of cell & gene therapy: Key trends to watch Through four infusions administered during one surgical procedure, Kebilidi is designed to address the underlying genetic cause of the disease by delivering a functional copy of the DDC gene directly to specific regions of the brain responsible for motor control – it was the first drug approved in the U.S. that is administered directly in the brain. The therapy uses an adeno-associated virus (AAV) vector to transport the gene into nerve cells, enabling them to produce the AADC enzyme that is missing in patients with the disorder. Once delivered, the new genetic instructions allow the cells to restore neurotransmitter production, helping to improve motor function and developmental outcomes. Because AADC deficiency requires intensive lifelong medical care and support, the economic burden on affected families and healthcare systems can be substantial. FDA officials hailed Kebilidi’s promise in a press release at the time, saying it represented “important progress” in treating debilitating genetic disorders. The agency also awarded PTC Therapeutics a rare pediatric disease priority review voucher, which the company sold for $150 million. Hemgenix Cost: $3.5 million Company: uniQure, CSL Behring Third on the list of the most expensive drugs is Hemgenix, developed by Amsterdam-based biotech uniQure in partnership with global biopharma CSL Behring. It is a one-time gene therapy for adults with hemophilia B, a rare, lifelong bleeding disorder caused by a single gene defect, resulting in insufficient production of factor IX – a protein primarily produced by the liver that helps to form blood clots. As a result, the disease makes people vulnerable to bleeding in their joints, muscles, and internal organs, which causes pain, swelling, and joint damage. Hemgenix reduces the rate of abnormal bleeding in people with hemophilia B by enabling the body to continuously produce factor IX. It is an AAV vector-based gene therapy, using an AAV5 vector to carry genetic DNA instructions to the liver, where factor IX proteins are then generated. These genetic instructions remain in the target cells but generally do not become a part of a person’s own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX. The therapy was approved in 2022 and was described as a “game changer” in hemophilia B treatment. Patients with hemophilia B typically require regular prophylactic infusions of factor IX, which becomes extremely expensive over the course of a lifetime. CSL Behring told Fierce Pharma at the time of approval that, despite the $3.5 million price tag, the drug would generate “significant cost savings for the overall healthcare system” and “significantly lower the economic burden of hemophilia B by reducing annual bleed rates, reducing or eliminating prophylactic therapy and generating elevated [factor] IX levels that last for years.” Elevidys Cost: $3.2 million Company: Sarepta Therapeutics When Sarepta Therapeutics’ Elevidys gained accelerated approval from the FDA in June 2023, it became the first gene therapy for the treatment of pediatric patients from four to five years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. An inherited degenerative disease that results in muscle weakness and loss of muscle tissue, DMD is the most common childhood form of muscular dystrophy and typically occurs in boys, affecting around one in 3,300. It worsens over time, and the average life expectancy is around 27 years. Elevidys is a one-time therapy designed to treat the underlying cause of DMD, which involves a dystrophin gene mutation that yields low levels of the dystrophin protein that is needed to strengthen muscle fibers. The gene therapy, therefore, works by delivering a gene that codes for a functional shortened dystrophin (called Elevidys micro-dystrophin) into the muscle tissue. According to BioPharma Dive, at the time of approval, Sarepta’s chief executive officer (CEO) Doug Ingram defended the $3.2 million price tag that made it one of the most expensive drugs in the world, saying that the price actually reflected a “conservative” approach to valuing the benefit to patients and their families of Elevidys, which Sarepta claimed would only be cost-effective at prices between $5 million and $13 million. Lyfgenia Cost: $3.1 million Company: Genetix Biotherapeutics Genetix Biotherapeutics (previously called bluebird bio), which has three of its products included in this list of most expensive drugs, won approval for Lyfgenia in December 2023. This made it the first gene therapy – along with Casgevy (which can be found further down the list) – to treat sickle cell disease. Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. The disease causes a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues. This mutation causes red blood cells to develop a crescent or “sickle” shape – hence the name. These sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage called vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). The recurrence of these events or crises can lead to life-threatening disabilities and/or early death. Although Lyfgenia was groundbreaking in that it was one of the first two gene therapies approved for sickle cell disease, the drug is priced around 40% higher than the list price of its competitor Casgevy. According to Fierce, at the time of approval, analysts from William Blair, RBC Capital Markets, Jefferies, Cantor Fitzgerald, and Barclays all said in separate notes to clients that the higher price may put Genetix at a disadvantage in the upcoming market competition. However, the company said that the price recognizes the “value the therapy may deliver through robust and sustained clinical benefits and the estimated lifetime impact that reducing or eliminating VOEs may have on patients’ healthcare utilization, future earnings, and life opportunities.” Skysona Cost: $3 million Company: Genetix Biotherapeutics In 2022, four weeks after Genetix Biotherapeutics won FDA approval for its gene therapy Zynteglo, the company also earned approval for Skysona. When Zynteglo was approved, it momentarily became the highest-priced drug in U.S. history at $2.8 million. But not for long, as Genetix decided to price Skysona even higher, making it available at a cost of $3 million. Chief commercial and operating officer Tom Klima justified the price, stating that it was a “data-driven” process that took into “consideration the positive clinical outcomes, quality of life improvements, health system cost savings, and potential societal impact of patients and families.” Skysona, which is another gene therapy, was approved as the first treatment to slow the progression of neurologic brain dysfunction in boys aged four to 17 years with early, active cerebral adrenoleukodystrophy (CALD). CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes irreversible, devastating neurologic decline, including major functional disabilities such as loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement. Nearly half of patients who do not receive treatment die within five years of symptom onset. Skysona is made from the patient’s own stem cells and is modified to contain a copy of the gene to make a functional enzyme called ALDP (adrenoleukodystrophy protein), whose production is prevented due to a mutation in the ABCD1 gene in people with CALD. This mutation prevents the breakdown of very long-chain fatty acids, ultimately resulting in the destruction of the sheath (myelin) that surrounds the nerve. Zynteglo Cost: $2.8 million Company: Genetix Biotherapeutics After Genetix won regulatory approval in Europe for its beta-thalassemia treatment Zynteglo in 2019, the company set the price of the treatment at €1.575 million ($1.8 million), making it one of the most expensive drugs in the region. But, unable to reach a consensus with authorities in Europe around fair gene therapy pricing, Genetix announced its departure from Europe in August 2021, calling the pricing situation “untenable” and the market “broken.” When Genetix then received FDA approval for Zynteglo in the U.S. in 2022, the market was more forgiving, allowing the company to price its gene therapy at $2.8 million. Although the price tag came as a shock to many, Genetix said that it works out cheaper than current beta-thalassemia treatment options. At the time of its approval, Zynteglo became the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. Beta-thalassemia is a type of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells in the blood through mutations in the beta-globin subunit, leading to insufficient delivery of oxygen in the body. Transfusion-dependent beta-thalassemia is the most severe form of the condition and generally requires lifelong red blood cell transfusions as the standard course of treatment, which is a lengthy process that patients typically undergo every two to five weeks. Zynteglo works by adding functional copies of a modified form of the beta-globin gene into a patient’s own hematopoietic stem cells. The gene is added into a patient’s cells ex vivo and is then infused into the patient, resulting in normal or near-normal levels of total hemoglobin and eliminating the need for regular red blood cell transfusions. Casgevy Cost: $2.2 million Company: Vertex Pharmaceuticals, CRISPR Therapeutics Casgevy’s approval by the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) in November 2023 marked the first time a CRISPR-based gene therapy had ever been given the green light. Shortly after, in December 2023, it was also approved by the FDA for the treatment of sickle cell disease, before the regulatory agency widened its U.S. approval in January 2024, stating that the therapy could also be used to treat transfusion-dependent beta-thalassemia. As a one-time gene therapy, Casgevy could prove to be a cure for patients. It works by turning on a gene called fetal hemoglobin, which people naturally have as fetuses, as well as when they are born. This gene prevents people with sickle cell disease or beta-thalassemia from experiencing symptoms until it turns off later in life. Therefore, Casgevy is designed to target a gene called BCL11A, which usually prevents the production of fetal hemoglobin in adults. It is administered by taking blood-producing stem cells from the bone marrow of people with sickle cell disease or beta-thalassemia, before using CRISPR/Cas9 to edit the genes encoding hemoglobin. Once the Cas9 enzyme reaches BCL11A, it cuts both DNA strands and disables the gene, before the modified stem cells are then infused back into the patient, giving rise to red blood cells containing fetal hemoglobin. Casgevy is priced at $2.2 million, but Vertex and CRISPR have said that, in the U.S., healthcare costs to manage transfusion-dependent beta-thalassemia are estimated to be more than $5 million over a person’s lifetime, making the one-time therapy cheaper in the long run. Gene therapies: The most expensive drugs in the world As evidenced by the list, gene therapies have broken all the records when it comes to the world’s most expensive drugs. And, as the biopharma industry has embraced treatments for orphan diseases – illnesses that typically affect a small population – we have seen more gene therapy approvals in the last few years, each one seemingly coming with an even more expensive price tag than the last. But it’s worth noting that the extreme rarity of the diseases that gene therapies are used to treat is exactly why the costs are so high. For example, the world’s most expensive drug, Lenmeldy, treats MLD, which only affects one in every 40,000 individuals in the U.S. This means that, even though they cost so much, selling gene therapies can be a tricky business for many companies. Genetix Biotherapeutics is a perfect example of this; once valued at more than $10 billion, the company sold itself for between $29 million and $45 million upfront to a pair of private equity firms in early 2025 after failing to build a market for its three innovative and expensive treatments. This article is reserved for subscribers Subscribe for free to continue reading.Enter your details to log in or subscribe. 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