Prognosis

Drug for Ultra Rare Disease Comes with Ultra High Price Tag

  • Givlaari’s net price will be $442,000 after discounts included
  • Medicine may treat 3,000 porphyria patients in U.S., Europe

Alnylam Pharmaceuticals headquarters in Cambridge, Massachusetts.

Photographer: Kristoffer Tripplaar/Sipa via AP
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Alnylam Pharmaceuticals Inc.’s Givlaari became the first drug to win U.S. approval for patients with an ultra-rare and devastating metabolic disorder. Shares jumped on the news.

It will come with an annual wholesale price tag of $575,000. Patients will need to take it for life.