Mar 21, 2024 - Health

Seaport pharma firm launches world's most expensive medicine

Illustration of text boxes in the shape of a hundred dollar bill and a pill clashing

Illustration: Sarah Grillo/Axios

A new gene therapy drug from a Boston-based company to treat a rare genetic disorder comes with a whopping price tag: $4.25 million.

Why it matters: The drug, known as Lenmeldy, is now the most expensive medicine in the world.

  • The Seaport's Orchard Therapeutics won approval for Lenmeldy Monday.

Around 40 children are born with the genetic disorder treated by Lenmeldy — metachromatic leukodystrophy — in the U.S. each year, according to CNN.

  • Makers of the new drug claim it can slow or even stop the progression of MLD with a single dose.

The bottom line: Instead of charging patients over $4 million, the price is what insurers, including public payers like Medicaid, will have to pay for the treatment.

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