Most Expensive Prescription Drugs in the U.S. Market
Many of the most expensive drugs target rare conditions and can cost patients and insurers millions of dollars each year

The rising costs of specialty medications are putting significant pressure on the U.S. healthcare system. Many of the most expensive drugs target rare conditions and can cost patients and insurers millions of dollars each year. Below are some of the leading examples:

  1. Zolgensma – A one-time gene therapy for spinal muscular atrophy, often topping the charts as the most expensive medicine in the USA with a price tag of over $2 million.
  2. Brineura – Used to treat CLN2 disease, with an annual price exceeding $700,000.
  3. Soliris – For PNH and aHUS, averaging $678,000 annually.
  4. Ravicti – Designed for urea cycle disorders, with a yearly Ravicti cost of around $600,000.
  5. Danyelza – Treats neuroblastoma, costing roughly $700,000 annually.
  6. Zokinvy – For Hutchinson-Gilford Progeria Syndrome, with treatment costs near $1 million a year.
  7. Myalept – Used for leptin deficiency, averaging more than $850,000 per year.
  8. Actimmune – An immune therapy with an Actimmune cost that can exceed $500,000 annually.
  9. Lumizyme – For Pompe disease, costing over $600,000 annually.
  10. Vumerity – A multiple sclerosis treatment, with costs up to $90,000 annually.
  11. Folotyn – A rare lymphoma therapy, with a monthly Folotyn price of about $120,000.
  12. Zilbrysq – New for generalized myasthenia gravis, with high costs and assistance programs.

These examples show how the most expensive medication market is evolving and how rare-disease therapies remain a major challenge for patients and insurers.

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