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US FDA approves Vertex’s new breakthrough therapy for cystic fibrosis

Published on 22/10/19 at 09:02am

The US FDA has approved Trikafta (elexacaftor/ivacaftor/tezacaftor) – the first triple combination therapy available for patients with most common cystic fibrosis mutation.

The drug approval comes five months ahead of schedule and just three months after Vertex submitted its regulatory application.

Trikafta is approved for patients 12 years and older with cystic fibrosis who have at least one F5080del mutation in the cystic fibrosis transmembrane conductance regulator gene, which is estimated to affect 90% of the cystic fibrosis population or roughly 6,000 people.

Vertex’s Chairman, President and Chief Executive Officer, Jeffrey Leiden, 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 U.S., 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 program 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 was generally well tolerated and demonstrated improvements in multiple outcome measures, including improvements in FEV1 , improvements in respiratory symptoms and in the 24 week study reduced the rate of pulmonary exacerbations as well as improvements in BMI.

Vertex is currently evaluating Trikafta in children aged 6 through to 11 with an ongoing phase 3 study.

Reshma Kewalramani, Executive Vice President and Chief Medical Officer at Vertex, added: “The incredible speed of this approval underscores our shared sense of urgency with the FDA and the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease.

“We remain committed to relentlessly pursuing the development of transformative therapies for all people living with this disease.”

Nik Kiran

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