‘Miracle’ Cystic Fibrosis Drug Kept Out of Reach in Developing Countries

About 90 percent of patients of Northern European ancestry have the most common mutation needed for the drug to work, compared with far fewer people from the Middle East, Asia and Africa. In India, estimates range from 19 to 44 percent.

Vertex is funding an academic project to better understand the genetics of cystic fibrosis patients in poorer countries. Dr. Milan Macek Jr., a geneticist in Prague, is working with doctors in lower-resourced countries to collect and analyze blood samples from willing patients. He has identified hundreds of diagnosed patients in Eastern Europe, the Middle East and Central Asia who have the most common mutation.

Belinda Nell, who is working on the action in South Africa, followed the news about Vertex’s drugs closely, as her two sisters grew increasingly frail with cystic fibrosis.

In 2014, Ms. Nell and her sister, Lorryn, who also had the disease, nursed a third sibling, Jennifer, as she was dying of it. Ms. Nell promised Lorryn that she would keep her from the same fate and, in early 2022, managed to obtain a couple months’ supply of Trikafta for her. But Lorryn’s lungs were too damaged, and she died last October.

“It’s vital that children everywhere get access from a young age so they don’t endure the end stage like I saw with my sisters,” Ms. Nell said.

Elif Ince contributed reporting from Istanbul.

Article source: https://www.nytimes.com/2023/02/07/health/cystic-fibrosis-drug-trikafta.html