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news.The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Vertex Pharmaceuticals’ triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), for treating cystic fibrosis (CF) in individuals aged six years and above.
CF leads to severe multi-organ dysfunction, predominantly affecting the lungs, and pancreas, among others. Credit: Robina Weermeijer on Unsplash.
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The approval is intended for those who have a minimum of one F508del mutation or another responsive CFTR gene mutation.
The company is working with the National Institute for Health and Care Excellence (NICE) and the National Health Service (NHS) to facilitate the treatment’s accessibility, which addresses the root cause of CF.
Following its approval by the US FDA last December for the same age group, Vertex is pursuing marketing authorisation with the European Medicines Agency (EMA) and regulatory bodies in Switzerland, Canada, New Zealand and Australia.
Vertex Pharmaceuticals Global Medicines Development and Medical Affairs executive vice-president and chief medical officer Carmen Bozic said: “For more than 20 years we have been focused on discovering medicines that treat the underlying cause of the disease with the goal of helping people live longer and better lives.
“The approval of Alyftrek, our fifth CFTR modulator regimen, represents another significant milestone in that journey for people with CF in the UK.”
CF is stated to be a rare genetic condition that leads to severe multi-organ dysfunction, predominantly affecting the lungs, pancreas, gastrointestinal (GI) tract and liver.
It is caused by mutations in the CFTR gene, which leads to defective or insufficient CFTR protein, leading to thick mucus accumulation and chronic lung infections.
According to the company, Alyftrek’s active components, vanzacaftor and tezacaftor, are tailored to upsurge the CFTR protein on the surface of the cell, aiding in protein processing and trafficking.
Deutivacaftor, as a potentiator, increases the channel open probability of the CFTR protein, enhancing salt and water flow through cell membranes.
