FDA Approves Orkambi for Children With CF Ages 1 to 2 Years

Orkambi® is now approved for use in children with cystic fibrosis ages 1-2 years who have two F508del mutations. This is the only modulator available to very young children with these mutations.

Sept. 2, 2022 | 3 min read

The U.S. Food and Drug Administration (FDA) approved Orkambi® (lumacaftor/ivacaftor) for children with cystic fibrosis ages 1-2 years who have two F508del mutations.

With this approval, more than 250 children are now eligible for Orkambi, and it is the only modulator therapy available to very young children with these mutations. Those 6 years and older with two F508del mutations are also eligible to take the modulators Symdeko® (tezacaftor/ivacaftor) and Trikafta® (elexacaftor/tezacaftor/ivacaftor). 

“Research indicates that starting treatment with modulators at a young age could help slow or even prevent the irreversible progression of cystic fibrosis, which is why Orkambi’s approval is great news for children now eligible to take a modulator for the first time,” said JP Clancy, MD, senior vice president of clinical research at the Cystic Fibrosis Foundation. “This is yet another step toward advancing our mission of ensuring we find viable treatments and ultimately a cure for all individuals with CF.” 

Modulators partially restore activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which maintains the proper balance of salt and fluids at the surface of the lungs and other organs. By restoring the protein’s activity, many downstream consequences of CF could be prevented, reducing the need for future chronic therapies. That is why the Foundation believes it is essential to start people with CF on modulators as early as possible to prevent lung damage and the onset of complications. 

This is the third expansion of Orkambi to younger age groups since Orkambi was first approved in 2015 for people with CF ages 12 years and older with two copies of the F508del mutation. The drug is one of four medicines approved to treat the faulty CFTR protein, the underlying cause of CF. 

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

Despite extraordinary progress in helping people with CF live longer and healthier lives, some people are either not eligible to take a modulator or they cannot tolerate the medication. There is still critical work to be done to help all people living with CF get a treatment for the underlying cause of their disease. To learn more about how we are funding research to find treatments for all people with CF, regardless of their mutations, visit Path to a Cure

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