New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most

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.

Lyfgenia will launch in the United States. Vertex has been prioritizing winning approval in six wealthy countries — the United States, Italy, Britain, France, Germany and Saudi Arabia — that, by one estimate, are home to 2 percent of the global sickle cell population.

Three-quarters of the world’s sickle cell patients are in sub-Saharan Africa. Several million of them are believed to be sick enough that they would be eligible for the new therapies...