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September 5, 2022

US FDA approves Vertex’s Orkambi for cystic fibrosis in children

The approval is based on a Phase III clinical trial of Orkambi in 46 children aged one to under two years.

The US Food and Drug Administration (FDA) has approved the expanded usage of Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to treat cystic fibrosis (CF) in children aged 12 months to under 24 months.

The treatment is indicated for CF patients who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 

Earlier, the therapy had obtained FDA approval for usage in CF patients aged two years and above with two F508del mutation copies.

The latest approval for use in children aged 12 to below 24 months is based on an open-label, multicentre Phase III clinical trial for 24 weeks.

It enrolled 46 children aged one to under two years with the F/F genotype. 

According to the findings, Orkambi was found to be well tolerated.

Additionally, the therapy’s safety profile and pharmacokinetics were in line with those seen in trials in subjects aged two years and above. 

Further trial data, including a decline in sweat chloride concentration, indicated the treatment’s potential for CF disease modification.

An oral therapy, Orkambi is a combination of lumacaftor and ivacaftor, which together help in hydrating and eliminating mucus from the airways.

With the latest development, nearly 300 children with two copies of the F508del mutation will receive the therapy for the first time to treat the underlying cause of the ailment.

Vertex Pharmaceuticals Global Medicines Development and Medical Affairs executive vice-president and chief medical officer Carmen Bozic said: “Treating children with cystic fibrosis as early in life as possible is critically important because early treatment has the potential to slow the progression of this devastating disease. 

“The approval is another important step on our journey to reach people of all ages living with cystic fibrosis who may benefit from our medicines.”

In July, the company entered a definitive agreement to acquire ViaCyte for $320m in cash.

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