Drug prices in the U.S. keep rising, and the biggest jumps are seen in specialty therapies and treatments for rare diseases. These medicines are often groundbreaking and essential, but their costs can be staggering. Many are counted among the most expensive drugs, with yearly costs running into the hundreds of thousands or even millions of dollars.

At the top of the list is Zolgensma, a gene therapy that targets spinal muscular atrophy. It’s given only once, but the price exceeds $2 million, making it the most expensive medicine in the USA. It has brought new hope to families, but its cost continues to raise debates about affordability and access.

Brineura is another high-priced therapy. Created for CLN2 disease, a rare neurological condition, it requires enzyme infusions directly into the brain and costs more than $700,000 annually. Soliris, used to treat PNH and atypical hemolytic uremic syndrome, comes in at about $678,000 each year. These prices reflect the difficulty of developing drugs for very small patient groups.

Ravicti, which treats urea cycle disorders, is critical for patients whose bodies cannot remove ammonia efficiently. The annual Ravicti cost is about $600,000, showing how rare conditions often come with high treatment expenses.

In oncology, price tags are also significant. Danyelza, used for high-risk neuroblastoma, costs close to $700,000 a year. Folotyn, a treatment for rare lymphoma, is even more striking. With a monthly Folotyn price of roughly $120,000, annual costs can easily pass $1 million. Such drugs are often the result of years of research and complex manufacturing.

Other rare-disease therapies are equally expensive. Zokinvy, designed for Hutchinson-Gilford Progeria Syndrome, can cost up to $1 million annually. Myalept, used to treat leptin deficiency and generalized lipodystrophy, has an average yearly cost of over $850,000. These drugs are essential to patients but usually require significant insurance coverage or assistance programs.

Immune and enzyme replacement therapies are another group of high-cost drugs. Actimmune, prescribed for chronic granulomatous disease and other immune conditions, has an Actimmune cost that can exceed $500,000 each year. Lumizyme, used for Pompe disease, also costs more than $600,000 annually. Such treatments are critical but remain financially out of reach for many without strong support systems.

Some specialty therapies are priced lower but are still significant investments. Vumerity, an oral medication for multiple sclerosis, averages about $90,000 per year. Though less costly than others on this list, it’s still a challenge for long-term care budgets.

A newer option, Zilbrysq, has entered the market to treat generalized myasthenia gravis. Pricing can vary depending on dosage and patient needs, but it aligns with other rare-disease drugs in terms of high overall cost.

These examples highlight the difficult balance between scientific progress and financial accessibility. The most expensive medication often offers transformative benefits, but price is a major barrier. Many patients rely on insurance, manufacturer programs, or nonprofit support to gain access.

As more therapies are developed, particularly in gene and cell therapy, the list of high-cost drugs will keep growing. Health systems and policymakers are exploring new payment models, including value-based pricing and outcomes-based contracts, to help manage these costs while ensuring patients get the care they need.

Keeping up with these changes is important for anyone impacted by rare diseases or high-cost treatments. The landscape of most expensive drugs is constantly evolving, and understanding these trends can help patients, families, and providers plan for the future.

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