Therapy Areas: Hereditary Disorders
Vertex Pharmaceuticals wins Health Canada's marketing authorization for TRIKAFTA to treat cystic fibrosis
21 June 2021 -

Biotechnology company Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported on Friday the receipt of Health Canada's marketing authorisation for the prescription medicine TRIKAFTA for the treatment of cystic fibrosis (CF) in people ages 12 years and older.

Cystic fibrosis (CF), a rare, life-shortening genetic disease affecting more than 80,000 people globally, is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.

Health Canada has approved TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation.

For the first time, approximately 1,100 eligible patients with CF ages 12 years and older who have at least one F508del mutation have a medicine that targets the underlying cause of their CF. TRIKAFTA is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface, added the company.

Additionally, TRIKAFTA's approval was supported by positive results of three global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF), a four-week Phase 3 study in 107 people with two F508del mutations (F/F) and a Phase 3 study in 258 people heterozygous for the F508del-CFTR mutation and a CFTR gating mutation (F/G) or a residual function mutation (F/RF), concluded the company.

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