New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most
December 11, 2023

(New York Times) – The Food and Drug Administration’s approval on Friday of two groundbreaking gene therapy treatments for sickle cell disease has brought a rare moment of hope and celebration to people with the agonizing blood disorder.
But there is no clear path for the new therapies — one-time treatments so effective in clinical trials that they have been hailed as cures — to reach the countries where the vast majority of people with sickle cell live. Shortly after the approval their manufacturers announced sticker prices in the millions of dollars: $3.1 million for Lyfgenia, made by Bluebird Bio, and $2.2 million for Casgevy, made by Vertex Pharmaceuticals. (Read More)