2020-11-29| Trials & Approvals

Vertex Bags EU Approval for Cystic Fibrosis Treatment in Children Aged 6-11 Years

by Daniel Ojeda
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On November 27, Vertex Pharmaceuticals announced that the label extension for the combination treatment of SYMKEVI (tezacaftor/ivacaftor) with KALYDECO (ivacaftor) has been approved by the European Commission. This treatment can now be used to treat cystic fibrosis in children 6-11 years who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, the most common mutation that leads to cystic fibrosis, or one copy of the F508del mutation and one copy of one of 14 mutations in the CFTR gene.

Cystic fibrosis (CF) is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which results in the production of a dysfunctional protein. CFTR protein is necessary to attract water to the surface of cells, without which the mucus in the lungs clogs the airway and traps bacteria. As a result, patients with CF suffer from infections, inflammation, respiratory failure, and progressive lung damage that have life-shortening consequences. The average life expectancy of a person with CF is around 37.5 years.

Tezacaftor addresses the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor enhances the function of the protein once it reaches the cell surface.

Globally, there are more than 75,000 people living with CF, with around 35,000 of them living in the United States. Currently, CF screening is performed in newborns and can be detected within the first month of life. Approximately 1,000 new cases are diagnosed each year. Before the creation of drugs that improved the function of the CFTR protein, the only treatment options were antibiotics for lung infections, anti-inflammatory treatments to reduce the swelling on the airways, mucus-thinning drugs.

“With this approval, children with CF in Europe ages 6 to 11 years with the most common mutation, F508del, have a new treatment option and children with certain residual function mutations will, for the first time, have a treatment option available that addresses the underlying cause of their CF,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “Today’s approval brings us closer to our ultimate goal of providing medicines for all people with CF.”

By Daniel Ojeda, Ph.D.

Related Article: An Outlook on the Evolution of Cystic Fibrosis Care



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