Potential new standard of care for cystic fibrosis nears approval

Cystic fibrosis is a rare, genetic disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands, and reproductive tract. It’s caused by certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which affects how much CFTR protein cells make and how well the protein works. People with cystic fibrosis have inherited two copies of a mutated CF gene – one copy from each parent.¹

The disease causes a build up of mucus. This can clog up the lungs and airways, making it harder to breathe. The median age of death is in the 30s, but advances in newborn screening, treatments, nutrition, and lung transplants help people live longer, healthier lives.

Treatment landscape and pipeline

Current therapies include symptom management and treatment of the underlying causes of the disease. CFTR modulator therapies help a faulty CFTR protein work properly.

The CFTR modulators developed so far are effective only in people with specific mutations.

Vanzacaftor/tezacaftor/deutivacaftor (Vertex Pharmaceuticals) is a next-in-class triple combination CFTR modulator pending FDA approval for treatment of cystic fibrosis in patients ages 6 and older who carry at least one copy of the most common gene mutation or other eligible mutations. It’s an oral, once-daily fixed-dose medicine that targets the root cause. Once-daily dosing might reduce barriers to successful treatment and increase adherence, especially among patients taking multiple medications.⁵

The anticipated FDA review date for vanzacaftor/tezacaftor/deutivacaftor is Jan. 2, 2025.⁶ If approved, it could become the standard of care for cystic fibrosis over market leader Trikaftra (elexacaftor-tezacaftor-ivacaftor) and other CFTR modulators, Kalydeco (ivacaftor) Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftors). Approved in 2019, Trikafta (also Vertex Pharmaceuticals), is taken twice daily with morning and evening doses.