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FDA approves Vertex's Trikafta in new indication, now potentially covering 90% of cystic fibrosis patients

Published on 23/10/19 at 10:49am

The FDA has granted marketing authorisation to Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF), it has emerged.

The approval relates specifically to patients over the age of 12 who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – the most common in the disease.

This means that around 6,000 additional patients will now have access to treatment for their condition. This is in addition to the approximately 12,000 patients who can already access one of Vertex’s three other FDA-approved CF therapies – this group will also be eligible to receive Trikafta. Altogether, this covers 90% of patients living with the condition. 

“Today’s approval is a historic moment in cystic fibrosis care, with the potential for more people to benefit from CFTR modulator therapy to treat the basic defect of their disease,” explained Dr Steven Rowe, Director at the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama. “In clinical trials, Trikafta was generally well tolerated and demonstrated improvements in multiple outcome measures in CF, including improvements in FEV1, improvements in respiratory symptoms and, in the 24-week F/MF study, a reduced rate of pulmonary exacerbations and improvements in BMI.”

Dr Jeffrey Leiden, Vertex's Chairman, President and Chief Executive Officer, also remarked: “Today marks a milestone for CF patients, their families and Vertex. After a 20-year journey together, we have received FDA approval of Trikafta: a single breakthrough medicine with the potential to treat up to 90% of all people with CF in the future. For approximately 6,000 people with CF in the US, Trikafta is the first medicine that can treat the underlying cause of their disease. I want to personally thank the hundreds of Vertex scientists who have been working on this programme for nearly 20 years – many of whom have dedicated their entire careers to changing the course of this disease; the CF Foundation which has provided support, encouragement and help throughout the journey; and most importantly the thousands of patients, caregivers, doctors and advocates who have courageously and persistently worked side-by-side with us to get to where we are today.”

Trikafta is currently awaiting review from the EMA, while Vertex is currently investigating the efficacy of the therapy in patients between the ages of six and eight years old with F/MF and F/F CF mutations. The company also confirmed it has plans to also look into its efficacy in children under the age of six in future.

Matt Fellows

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