Kalydeco now approved in US for children as young as 1 month old

First and only CFTR modulator approved for this age group

Steve Bryson, PhD avatar

by Steve Bryson, PhD |

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The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for children with cystic fibrosis (CF) as young as one month old, who have at least one gating mutation in their CFTR gene that is responsive to the oral treatment.

The agency’s approval was supported by data from the now-completed 24-week Phase 3 ARRIVAL trial (NCT02725567), which evaluated the therapy’s safety, pharmacological properties, and preliminary efficacy in 56 CF patients who were younger than 24 months.

Its safety profile was similar to that seen in older children and adults, according to Vertex Pharmaceuticals, the company that markets Kalydeco.

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Kalydeco first approved in US in 2012

First approved in the U.S. in 2012, and prior to this label expansion, the treatment was available in the U.S., Canada, and the European Union for older CF patients, starting at 4 months old, with certain CFTR gene mutations.

This latest approval to include even younger patients means Kalydeco is the first and only CFTR modulator approved for this age group.

“As a physician caring for infants and children with cystic fibrosis, I see the importance of initiating therapies early in life that may slow disease progression,” Margaret Rosenfeld, MD, one of ARRIVAL’s principal investigators, from Seattle Children’s Research Institute and department of pediatrics at the University of Washington School of Medicine, said in a press release.

“Today’s approval provides many families and caregivers comfort in knowing that there is a highly effective modulator therapy available for their babies with CF,” Rosenfeld said.

CFTR is a protein channel that acts like a gate, controlling the movement of chloride salts at the cell membrane. CF is caused by mutations in the CFTR gene that encodes the channel, leading to defective or missing CFTR protein. As a result, thick and sticky mucus builds up in various organs.

As part of the CFTR modulator class of medications, Kalydeco is referred to as a CFTR potentiator because it keeps the CFTR gate open for longer at the cell surface. This improves the flow of salt and the clearance of mucus. It was the first medicine approved to treat the underlying cause of CF in people with certain mutations.

As a physician caring for infants and children with cystic fibrosis, I see the importance of initiating therapies early in life that may slow disease progression.

Kalydeco approved multiple times in US for different age groups

The FDA expanded its approval of Kalydeco multiple times, including for the treatment of children 1 to 2 years old in 2018, and in 2019 to cover children as young as 6 months. More recently, Kalydeco was approved for infants 4 months and older with CF caused by specific CFTR mutations. Whether the treatment is safe and effective in children younger than 1 month is not known.

“Treating the underlying cause of cystic fibrosis as early as possible is important, and this approval, the first for a CFTR modulator in this age group, means families will now have a medicine for eligible infants,” said Carmen Bozic, MD, Vertex’s chief medical officer and executive vice president of global medicines development and medical affairs.