CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001.
The CEO of Vertex Pharmaceuticals is to give evidence before influential UK politicians on Thursday, who are investigating the ongoing row over the price of cystic fibrosis drugs in England.
Orkambi was initially approved in Europe in 2015 for patients with CF ages 12 years and older who have two copies of the F508del mutation and was later expanded to include children ages six to 11 years.