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Triple therapy approved to treat cystic fibrosis
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The FDA announced it has approved elexacaftor/ivacaftor/tezacaftor, the first triple combination therapy for patients with the most common cystic fibrosis mutation.
The agency approved the therapy (Trikafta, Vertex Pharmaceuticals) for patients aged 12 years and older with cystic fibrosis who have one or more F508del mutations in the CFTR gene, according to a press release.
This population accounts for approximately 90% of people who have cystic fibrosis, the agency stated in the release.
The agency had granted the therapy priority review and gave it fast track, orphan drug and breakthrough therapy designations. Vertex will receive a rare pediatric disease priority review voucher for developing the therapy, according to the release.
“At the FDA, we’re consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review. Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy,” Ned Sharpless, MD, acting commissioner of the FDA, said in the release. “In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients’ quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options. That’s why we used all available programs, including priority review, fast track, breakthrough therapy and orphan drug designation, to help advance today’s approval in the most efficient manner possible, while also adhering to our high standards. The FDA remains committed to advancing novel treatment options for areas of unmet patient need, particularly for diseases affecting children.”
Approval was based on two trials that showed elexacaftor/ivacaftor/tezacaftor increased percent predicted FEV1 compared with placebo or ivacaftor/tezacaftor, according to the release.
Rash and influenza events occurred more often in patients assigned elexacaftor/ivacaftor/tezacaftor than in those assigned placebo, the agency stated in the release.
Disclosure: Sharpless is an employee of the FDA.