The cost of specialty treatments continues to climb, creating serious challenges for the U.S. healthcare system. Many of the most expensive drugs are developed for rare diseases, and some reach millions of dollars annually. Below is an updated list of some of the highest-priced options:

1. Zolgensma – A one-time gene therapy for spinal muscular atrophy, leading the market as the most expensive medicine in the USA at over $2 million.

2. Brineura – For CLN2 disease, costing more than $700,000 each year.

3. Soliris – Treats PNH and aHUS with an average annual cost of $678,000.

4. Ravicti – Used for urea cycle disorders, with a yearly Ravicti cost around $600,000.

5. Danyelza – Neuroblastoma therapy priced at roughly $700,000 annually.

6. Zokinvy – For Hutchinson-Gilford Progeria, costing up to $1 million per year.

7. Myalept – Addresses leptin deficiency at over $850,000 annually.

8. Actimmune – Immune therapy with an Actimmune cost surpassing $500,000 annually.

9. Lumizyme – Pompe disease therapy costing more than $600,000 each year.

10. Vumerity – Multiple sclerosis drug, reaching about $90,000 annually.

11. Folotyn – Rare lymphoma treatment with a monthly Folotyn price close to $120,000.

12. Zilbrysq – For generalized myasthenia gravis, with high treatment costs and assistance programs.

These therapies show how the most expensive medication market continues to grow, putting financial strain on patients, providers, and payers.

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