The 12 Most Expensive Medications in U.S. Healthcare

Drug prices in the United States continue to rise, but the sharpest increases are seen with treatments for rare diseases and complex conditions. These medicines are often life-changing or even lifesaving, but they come with very high costs because of the research, manufacturing, and small patient populations they serve. Many of them are counted among the most expensive drugs, and their prices are reshaping healthcare spending.

Leading the pack is Zolgensma, a gene therapy that treats spinal muscular atrophy in young children. It’s a single-dose treatment, but the price exceeds $2 million, making it the most expensive medicine in the USA. The therapy offers remarkable results for patients, yet its cost limits accessibility and challenges insurance systems.

Another costly option is Brineura, created for CLN2 disease, a rare neurological condition. Brineura is administered through brain infusions and costs more than $700,000 each year. Soliris, which treats PNH and aHUS, is another high-cost drug, with annual spending around $678,000. Both are vital to patients who need them, but their prices highlight the challenges of developing therapies for rare illnesses.

Ravicti is used to treat urea cycle disorders, helping to manage ammonia levels that can become toxic. It’s critical for patients, but the yearly Ravicti cost is about $600,000. This expense reflects not only the difficulty of producing rare-disease medications but also the importance of these drugs to those who rely on them daily.

Cancer therapies often carry some of the highest prices. Danyelza, for children with high-risk neuroblastoma, costs close to $700,000 per year. Folotyn, which treats a rare form of lymphoma, can cost about $120,000 each month, meaning the annual Folotyn price can reach or exceed $1 million. Such high prices reflect the research and precision needed to create effective cancer treatments.

Other rare-disease therapies are also extremely expensive. Zokinvy, developed for Hutchinson-Gilford Progeria Syndrome, is close to $1 million per year. Myalept, used to treat leptin deficiency and generalized lipodystrophy, costs around $850,000 annually. For families facing these conditions, these drugs are critical but often require significant financial help.

Immune and enzyme therapies are another category of costly treatments. Actimmune, prescribed for chronic granulomatous disease and other immune issues, has an Actimmune cost that can exceed $500,000 each year. Lumizyme, used for Pompe disease, also has a price tag above $600,000 annually, reflecting its role as a lifelong therapy.

Some medications, while not in the million-dollar range, still represent significant expenses. Vumerity, an oral drug for multiple sclerosis, costs roughly $90,000 per year. Though lower than the others mentioned, it still adds up over years of treatment.

Zilbrysq, approved for generalized myasthenia gravis, is one of the newer entrants to this high-cost market. While exact prices vary, it reinforces the pattern of rising costs for rare-disease treatments and the need for patient assistance programs.

These examples demonstrate the tension between medical innovation and affordability. The most expensive medication often represents breakthroughs in treatment but remains financially out of reach for many. Patients and families depend heavily on insurance, copay support, or manufacturer programs to gain access.

The future will likely see even more of these therapies. As gene and cell therapies expand, prices are expected to remain high, and health systems are exploring new approaches such as value-based contracts and outcome-based pricing to balance innovation with sustainability.

Staying informed about these costs is essential for patients, providers, and policymakers. The list of most expensive drugs changes as new treatments are approved, and tracking these trends can help people prepare for the challenges ahead.

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