The 12 Most Expensive U.S. Medications in 2025

The cost of innovative medicines in the U.S. continues to rise, especially for treatments developed for rare and complex diseases. These therapies often deliver life-changing or life-saving results, but they also represent some of the most expensive drugs available today. Some treatments reach annual costs in the millions, putting enormous pressure on patients, insurers, and healthcare systems.

Zolgensma, a breakthrough gene therapy for spinal muscular atrophy, is a clear example. Delivered as a single dose, it comes with a price tag of more than $2 million, earning the title of the most expensive medicine in the USA. While its clinical impact is remarkable, its cost raises ongoing questions about access and affordability.

Brineura is another high-cost treatment. It’s used for CLN2 disease, a rare degenerative condition, and requires regular infusions directly to the brain. The cost exceeds $700,000 per year. Soliris, prescribed for PNH and aHUS, costs about $678,000 annually, illustrating how drugs for rare diseases often command very high prices.

Ravicti addresses urea cycle disorders, a condition that prevents the body from properly removing ammonia. The yearly Ravicti cost is around $600,000, making it essential but financially challenging for many families.

In cancer treatment, costs are equally steep. Danyelza, for high-risk neuroblastoma, costs roughly $700,000 a year. Folotyn, designed for certain rare lymphomas, is even more striking. At nearly $120,000 per month, the annual Folotyn price can top $1 million, showing how specialized cancer drugs can strain even strong insurance plans.

Other rare-disease drugs follow similar trends. Zokinvy, for Hutchinson-Gilford Progeria Syndrome, costs close to $1 million a year. Myalept, used to treat leptin deficiency and lipodystrophy, has an average annual price over $850,000. Such treatments are vital but usually require extensive support through insurance or assistance programs.

Immune therapies and enzyme replacements also rank among the most expensive. Actimmune, prescribed for conditions like chronic granulomatous disease, has an Actimmune cost that can exceed $500,000 each year. Lumizyme, used for Pompe disease, similarly costs more than $600,000 annually.

Some treatments fall below these numbers but are still significant investments. Vumerity, for multiple sclerosis, comes in at about $90,000 annually. Over a lifetime, costs add up quickly.

Newer entrants like Zilbrysq, a therapy for generalized myasthenia gravis, also demonstrate how rare-disease treatments continue to demand high prices. While the exact cost varies, it follows the same trend of premium pricing.

These examples highlight the tension between innovation and affordability. The most expensive medication can provide transformative results, but it often comes with significant financial barriers. Many patients depend on health insurance, copay assistance, and manufacturer programs to gain access to these drugs.

As the pharmaceutical industry develops more gene therapies and advanced biologics, the list of most expensive drugs will continue to grow. Healthcare providers and policymakers are exploring models like outcomes-based pricing and payment plans to balance innovation and cost.

For patients, caregivers, and professionals, staying informed about these high-cost treatments is crucial. Understanding the trends and challenges around the most expensive drugs helps in planning, advocacy, and accessing support.

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