Source:

Press Release.

Disclosures: Kewalramani is CEO and president of Vertex Pharmaceuticals. Laguna reports no relevant financial disclosures.
June 09, 2021
1 min read
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FDA approves Trikafta for children aged 6 to 11 with cystic fibrosis

Source:

Press Release.

Disclosures: Kewalramani is CEO and president of Vertex Pharmaceuticals. Laguna reports no relevant financial disclosures.
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The FDA has expanded use of elexacaftor/tezacaftor/ivacaftor for children aged 6 to 11 years with cystic fibrosis with certain mutations, according to a press release from Vertex Pharmaceuticals.

The new approval includes children who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive to elexacaftor/tezacaftor/ivacaftor (Trikafta) based on in vitro data.

FDA approval
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This therapy was previously approved by the FDA for use in individuals with cystic fibrosis aged 12 years and older with at least one copy of the F508del mutation that is responsive in vitro.

An additional dosage strength is now available — elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg) in connection with this approval, according to the release.

“Today’s approval is a critical milestone in our efforts to deliver medicines that help treat the underlying cause of this devastating disease as early in life as possible,” Reshma Kewalramani, MD, CEO and president of Vertex Pharmaceuticals, said in the release. “We can now reach approximately 1,500 newly eligible children in the U.S., and we continue to pursue approval for this expanded indication in other countries.”

The expanded approval was based on a 24-week, phase 3, open-label, multicenter study that included 66 children aged 6 to 11 years with cystic fibrosis and either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. Results showed that triple therapy was well tolerated, with similar safety data as observed in previous studies of individuals aged 12 years and older. The data were published in the American Journal of Respiratory and Critical Care Medicine.

“I look forward to now being able to treat younger patients with this breakthrough medicine, including those who have not presented major signals of disease progression,” Terri Laguna, MD, MSCS, associate director of the Cystic Fibrosis Center and division head of pulmonary and sleep medicine at Ann & Robert H. Lurie Children’s Hospital of Chicago, said in the release. “In addition to bringing Trikafta to a younger patient population, patients not previously eligible for any CFTR modulator will now be able to access a treatment that targets the underlying cause of their disease.”

Reference:

Burgel PR, et al. Am J Respir Crit Care Med. 2021;doi:10.1164/ajrccm.202011-4153OC.

Editor’s note: This article was updated on June 10, 2021, to correctly describe the additional dosage strength now available.