FDA Approves Kalydeco for Infants as Young as 4 Months

The U.S. Food and Drug Administration has approved the use of Kalydeco® (ivacaftor) for infants with cystic fibrosis as young as four months who have certain mutations.

Sept. 25, 2020 | 3 min read

The U.S. Food and Drug Administration (FDA) has approved the use of Kalydeco® (ivacaftor) for infants with cystic fibrosis as young as four months who have certain mutations.

Highly effective CFTR modulators, including Kalydeco, are known to have a transformative effect on the health and wellbeing of people with CF, but these therapies do not reverse established lung damage. Research shows that starting treatment with a modulator at a young age could help slow or even prevent the irreversible progression of cystic fibrosis, dramatically altering the course of disease over time.

“We believe that people with CF will experience the maximum benefit from these therapies when they begin treatment early in life, before the disease has caused significant damage,” said JP Clancy, Vice President of Clinical Research, Cystic Fibrosis Foundation. “Our hope is that those who start on modulators as infants may never experience many of the classic symptoms of CF, and we are committed to supporting ongoing research to better understand the impact of early treatment.” 

During a 24-week study in six children between 4 and 6 months, the safety profile of Kalydeco was similar to that observed in older children and adults.

Kalydeco was initially approved in 2012 for treatment of people ages 6 and older with the gating mutation G551D, who make up 4 percent of the 30,000 people with CF in the U.S. Last year, approval was expanded to babies ages 6 months and older with one of 38 mutations.

It was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific, and financial support from the Cystic Fibrosis Foundation.

For more information about today's announcement, see the Vertex press release.

Despite extraordinary progress in helping people with CF live longer and healthier lives, there is still critical work to be done to help all people living with this disease, including those who won't benefit from modulators such as Kalydeco. Through its Path to a Cure, the CF Foundation has dedicated $500 million to develop new treatments for the underlying cause of the disease and, one day, a cure.

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