In the U.S., the price of advanced therapies continues to rise, especially for rare and complex conditions. These treatments save and extend lives but often carry staggering price tags. Some are among the most expensive drugs ever developed, with annual costs reaching hundreds of thousands or even millions of dollars.
A clear example is Zolgensma, a one-time gene therapy for spinal muscular atrophy. Its cost is over $2 million, earning it the title of the most expensive medicine in the USA. While it offers a potential cure for young children, the price remains a major concern for families and insurers.
Another high-cost therapy is Brineura, which treats CLN2 disease. Administered directly into the brain, it exceeds $700,000 per year. Similarly, Soliris, used for PNH and aHUS, comes in at around $678,000 annually.
Ravicti, used for urea cycle disorders, is another major expense. It helps prevent toxic ammonia buildup, but the yearly Ravicti cost hovers around $600,000.
Cancer treatments also dominate the upper price range. Danyelza, which treats high-risk neuroblastoma, is priced near $700,000 annually. Folotyn, a therapy for rare lymphomas, costs about $120,000 monthly, which means the annual Folotyn price can exceed $1 million.
Other specialty therapies follow similar trends. Zokinvy, created for Hutchinson-Gilford Progeria Syndrome, is close to $1 million per year. Myalept, for lipodystrophy and leptin deficiency, averages about $850,000 annually.
Immune system and enzyme replacement therapies are also extremely costly. Actimmune, used for conditions like chronic granulomatous disease, has an Actimmune cost exceeding $500,000 annually. Lumizyme, for Pompe disease, is another drug with yearly costs surpassing $600,000.
While not as high, some therapies still require significant spending. Vumerity, for multiple sclerosis, is roughly $90,000 annually.
Zilbrysq, a newer drug for generalized myasthenia gravis, is another example of rare-disease treatments contributing to these rising expenses.
These examples highlight the tension between medical innovation and financial accessibility. The most expensive medication often offers groundbreaking benefits, but cost remains a barrier for many. Insurance, assistance programs, and pharmaceutical support are essential for access.
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