A Look at the 12 Highest-Priced Drugs in America

Posted 2025-08-26 08:03:44

The cost of prescription medications in the United States has been steadily increasing, but nowhere is this more evident than in the market for specialty therapies and rare-disease treatments. These drugs are often designed for small patient populations, involve complex manufacturing, and require significant research and development. As a result, they carry price tags that would have been unimaginable a decade ago. Many of these therapies are now classified among the most expensive drugs, placing enormous financial strain on healthcare systems, insurance providers, and patients.

At the top of this list is Zolgensma, a gene therapy for spinal muscular atrophy. Unlike most treatments, Zolgensma is administered only once, but that single dose comes with a price tag of more than $2 million. Its effectiveness in treating such a devastating condition has made it a breakthrough therapy, but its cost keeps it firmly in place as the most expensive medicine in the USA.

Other high-cost therapies include Brineura, used to treat CLN2 disease. Brineura’s annual cost is more than $700,000, largely because the disease it targets is extremely rare and requires an enzyme replacement therapy delivered directly to the brain. Similarly, Soliris, an established therapy for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), has a yearly cost of about $678,000. These therapies highlight a broader pattern: when treatments are developed for ultra-rare conditions, the lack of a large patient base makes affordability a challenge.

Ravicti is another therapy that frequently appears on these lists. Designed to treat urea cycle disorders, Ravicti’s annual Ravicti cost hovers around $600,000. The drug helps patients manage ammonia levels in the blood, which, if left untreated, can lead to life-threatening complications. Despite its high price, Ravicti remains a critical therapy for those who depend on it.

Cancer therapies are another major contributor to rising drug costs. Danyelza, developed for the treatment of high-risk neuroblastoma, comes in at roughly $700,000 annually. Likewise, Folotyn, used for a rare type of lymphoma, can cost patients nearly $120,000 per month, making its Folotyn price one of the highest among oncology treatments. These drugs often require complex production methods and years of clinical testing, factors that contribute to their expense.

The list also includes therapies for genetic and metabolic conditions. Zokinvy, created to treat Hutchinson-Gilford Progeria Syndrome, costs nearly $1 million annually. Myalept, a therapy for generalized lipodystrophy and leptin deficiency, averages about $850,000 each year. Both drugs are essential to managing serious, life-shortening conditions but remain financially out of reach for many without insurance or assistance.

Immune and enzyme replacement therapies also feature prominently. Actimmune, which helps boost immune function in conditions like chronic granulomatous disease, carries an Actimmune cost that can exceed $500,000 annually. Lumizyme, a therapy for Pompe disease, requires ongoing infusions and has an annual cost above $600,000. Each of these treatments illustrates how innovation often comes with significant expense.

Not all specialty medications are in the million-dollar range, but many still represent substantial financial burdens. For example, Vumerity, an oral therapy for multiple sclerosis, costs about $90,000 per year, which is lower compared to other drugs on this list but still significant for long-term care.

A newer addition to the market, Zilbrysq, is used to treat generalized myasthenia gravis. While pricing can vary depending on dosing and assistance programs, it remains among the newer high-cost options.

These drugs highlight the ongoing tension between medical innovation and affordability. On one hand, these therapies offer hope to patients with conditions that were once untreatable. On the other hand, their prices often make access difficult and place stress on insurance plans and government health programs. Patients and caregivers often need support from copay assistance, manufacturer programs, or non-profit organizations to manage costs.

The landscape of expensive therapies is also evolving. New gene and cell therapies are entering the market every year, and each one brings the possibility of transformative care at a high price. Policymakers and healthcare organizations are debating strategies like value-based pricing, risk-sharing agreements, and expanded access programs to help make these drugs more sustainable.

For those following the market, keeping track of pricing trends is critical. Whether you are interested in learning about cutting-edge gene therapies or enzyme replacements, understanding where costs stand today is essential for planning and policy. The complete list of the most expensive medication is always changing as new treatments are approved and pricing models evolve.

These examples show that while innovation in medicine saves lives, it also comes with significant financial challenges. For patients, providers, and payers alike, the balance between access and cost will remain a major focus.

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