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2022-09-05| Trials & Approvals

Vertex’s Orkambi Racks Up Another Approval For Cystic Fibrosis

by Joy Lin
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Vertex has announced the US FDA approval of Orkambi (lumacaftor/ ivacaftor) to include children with cystic fibrosis (CF) ages 1 to 2 years with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 

Orkambi was first approved to treat cystic fibrosis in 2015 in the US. The drug is now marketed in more than 30 countries. 

Related article: Verve and Vertex Agree to Co-Develop In Vivo Gene Editing Program for Liver Disease

Clearing The Airways With Orkambi 

Cystic fibrosis is a rare genetic disease affecting more than 83,000 people globally. CF is caused by mutations in the CFTR gene, which codes for a surface protein that enables salt and water to flow into and out of the cells of multiple organs. The F508 deletion in CFTR results in a misfolded protein, which in turn leads to defective proteins, or an absence of proteins. As cells lose their ability to transport salt and water across their membrane, the body suffers a chronic buildup of sticky mucus in the airways and progressive lung damage. 

Orkambi is an oral drug that contains the active ingredients lumacaftor and ivacaftor. Lumacaftor is designed to increase the amount of functioning CFTR protein on the cell surface by acting as a scaffold that can help the misfolded protein fold and route correctly. Ivacaftor is designed to facilitate CFTR proteins in transporting salt and water across the cell membrane. The combined action of the two helps hydrate and clear mucus from the airways. 

The expanded approval for Orkambi is based on a Phase 3 study in 46 children with CF aged 1 to less than 2 years old. The drug was well-tolerated and the pharmacokinetics profile was similar to that observed in studies with older patients. 

“Treating children with cystic fibrosis as early in life as possible is critically important, because early treatment has the potential to slow the progression of this devastating disease,” said Carmen Bozic, M.D., Chief Medical Officer of Vertex. 

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