Specialty drug prices continue to rise, putting more pressure on the U.S. healthcare system. Many of the most expensive drugs are for rare conditions, with some treatments costing millions each year. Below is a list of the top medications and their approximate costs:
Zolgensma – Gene therapy for spinal muscular atrophy, often considered the most expensive medicine in the USA at over $2 million per treatment.
Brineura – Treats CLN2 disease with annual costs exceeding $700,000.
Soliris – For PNH and aHUS, this therapy costs about $678,000 annually.
Ravicti – For urea cycle disorders, the yearly Ravicti cost is around $600,000.
Danyelza – Treats neuroblastoma, costing roughly $700,000 a year.
Zokinvy – For Hutchinson-Gilford Progeria Syndrome, annual costs can reach $1 million.
Myalept – For leptin deficiency, averaging more than $850,000 annually.
Actimmune – Immune therapy with an Actimmune cost that can exceed $500,000 annually.
Lumizyme – For Pompe disease, costing more than $600,000 annually.
Vumerity – A multiple sclerosis treatment costing about $90,000 per year.
Folotyn – Rare lymphoma therapy with a monthly Folotyn price of nearly $120,000.
Zilbrysq – Recently approved for generalized myasthenia gravis, with high costs and support programs.
These examples show how the market for rare-disease therapies is driving up the cost of the most expensive medication and challenging patients, providers, and insurers alike.
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