Vimizim launch price annual 2014 $380000?

Yes—Vimizim (galsulfase), the enzyme‑replacement therapy for mucopolysaccharidosis type VII (Sly syndrome), was launched in 2014 and the U.S. launch price was cited at roughly $380 000 per year (about $1 200 / mg, or $3 400 / kg of body weight).

Quick background

Why the price is high

1. Rare‑disease status – The drug is approved under the Orphan Drug Act, allowing a higher price to recoup development costs for a very small patient population (estimated 40‑50 U.S. patients).
2. Production complexity – Galsulfase is a recombinant enzyme produced in CHO cells; the manufacturing process is tightly controlled and costly.
3. Long‑term therapy – Treatment is lifelong and must be continued weekly, so the cost accumulates annually.

Current context (post‑2014)

• Price adjustments – Prices have fluctuated somewhat since launch due to payer negotiations, rebates, and formulary placement.
• Insurance coverage – Most U.S. payers require prior authorization; coverage decisions often hinge on patient‑specific criteria (e.g., baseline organ function).
• Patient support – Shire/Takeda offers a patient assistance program to help offset costs for eligible patients.

If you need more detail

• Clinical efficacy – Vimizim slows progression of cardiac, liver, and skeletal disease but does not cure the disorder.
• Safety profile – Infusion‑related reactions are the most common adverse events; pre‑medication with antihistamines or steroids is frequently used.

Feel free to ask if you’d like more specific information—such as dosing for a particular patient weight, latest payer policies, or data on clinical outcomes.