Vertex Pharmaceuticals Inc. (Nasdaq: VRTX) has won marketing authorization for the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children with cystic fibrosis who are between the ages of 6 and 11 years old.
The latest approval covers children with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The approval allows an additional dosage strength of Trikafta tablets consisting of elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg, and ivacaftor 75 mg.
According to the company, the new approval will provide a treatment option to roughly 500 children.
“This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF,” said Dr. Reshma Kewalramani, chief executive officer and president at Vertex. “We will now work closely with all provinces and territories to secure access for eligible patients as quickly as possible.”
Before winning the new approval, Vertex conducted a 24-week Phase 3 open-label, multicenter study enrolling 66 children between 6 and 11.