EC approves Vertex’s Kalydeco to Treat Infants With Cystic Fibrosis

EC approves Vertex’s Kalydeco to Treat Infants With Cystic Fibrosis

By, Admin 30 April 2024

European Commission approves Vertex’s Kalydeco to treat infants with cystic fibrosis ages 1 month and older

Overview

Vertex Pharmaceuticals announced that the European Commission has granted approval for the label expansion of Kalydeco (ivacaftor) for the treatment of infants down to 1 month of age with cystic fibrosis (CF) who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

Words from CMO: Vertex

“Today’s approval is an important milestone for the cystic fibrosis community. Treating CF early in life can potentially slow the progression of the disease, which is why it is so important to start treatment from a very young age,” said Carmen Bozic, M.D., executive vice president, global medicines development and medical affairs, and chief medical officer, Vertex.

Access for Kalydeco (ivacaftor)

  • As a result of existing access agreements in Austria, Czech Republic, Denmark, Ireland, Norway, Sweden, and The Netherlands, eligible patients will have access to the expanded indication of Kalydeco (ivacaftor) shortly following regulatory approval by the European Commission. 
  • Vertex will continue to work with reimbursement authorities across the European Union to ensure access for all other eligible patients. 
  • In the UK, following MHRA approval at the end of 2023, and as a result of the existing reimbursement agreement between Vertex and the National Health Service, eligible infants ages 1 month and older in the UK have access to this expanded indication for Kalydeco (ivacaftor).

Cystic Fibrosis

  • Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. 
  • CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. 
  • CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. 
  • Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. 
  • While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. 
  • CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. 
  • The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. 
  • In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. 
  • The median age of death is in the 30s, but with treatment, projected survival is improving.

Mutations in the CFTR Gene

  • In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. 
  • Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. 
  • Kalydeco (ivacaftor) was the first medicine to treat the underlying cause of cystic fibrosis in people with specific mutations in the CFTR gene.

About Kalydeco

Kalydeco (ivacaftor) is a prescription medicine for the treatment of people with CF aged at least 1 month and weighing at least 3 kg who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: 
R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

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