The U.S. Food and Drug Administration (FDA) has approved the new triple-combination modulator Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) for people with cystic fibrosis ages 6 and older who have a mutation that is eligible for Trikafta or one of 31 rare mutations that had not been approved previously for a modulator.  

The FDA approved Alyftrek based on results from clinical trials comparing the drug to Trikafta, as well as lab test data that showed additional rare mutations were responsive to Alyftrek. [Trikafta was approved for 94 additional rare mutations earlier today.]

Alyftrek offers another option for people with CF, including those who cannot tolerate Trikafta. In addition, Alyftrek only needs to be taken once a day — compared to twice a day for other modulators — potentially making the dosing regimen easier for people with CF.  

“We are excited that there is another treatment option for people with cystic fibrosis, especially for those who previously weren’t eligible for a modulator,” said JP Clancy, MD, senior vice president of clinical research for the Cystic Fibrosis Foundation. “While we continue to support the development of newer and better modulators, we are also aggressively pursuing the advancement of genetic therapies, prioritizing developing treatments for people not benefiting from modulators.” 

Clinical trial results for Alyftrek showed improvements in lung function comparable to those seen with Trikafta, and a reduction in sweat chloride levels that exceeded treatment with Trikafta, according to Vertex Pharmaceuticals, which makes both drugs. Because the Foundation is aware of published reports of mental and cognitive side effects of people with CF on Trikafta, it is funding a study in development that will examine mental health and cognitive function of people with CF who start Alyftrek. Alyftrek comes with a boxed warning on the risk of liver injury and liver failure. Individuals interested in taking Alyftrek should speak with their care team with any questions or concerns.

People with CF who currently qualify for Trikafta as well as those who have one of the following rare mutations of the CFTR gene have been approved for Alyftrek:  

If you have questions navigating insurance coverage, get help by calling Compass at 844-COMPASS (1-844-266-7277) to speak with a case manager. You can also email compass@cff.org.