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The FDA has expanded indications for two targeted combination treatments, Alyftrek and Trikafta, that will now benefit more individuals with cystic fibrosis, according to a press release from the drugs’ manufacturer Vertex Pharmaceuticals.
Both medications address the underlying cause of cystic fibrosis by targeting the defective cystic fibrosis transmembrane conductance regulator (CFTR) protein and enabling it to function properly. New clinical and in vitro data support the effectiveness of the medications for approximately 500 additional CFTR genetic variants, which expands the eligible treatment population to approximately 95% of cystic fibrosis patients in the United States, according to the company.
Alyftrek, a vanzacaftor/tezacaftor/deutivacaftor combination, was initially approved by the FDA in 2024 for cystic fibrosis treatment in patients aged 6 years and older with a more limited range of CFTR mutations. The additional indication extends the drug’s use to patients aged 6 years and older with any CFTR variant that shows response based on clinical data, in vitro data, or demonstrated production of CFTR protein.
Trikafta, a combination of elexacaftor/tezacaftor/ivacaftor, was initially approved by the FDA in 2019, with additional indications in 2024 to include patients aged 2 years and older with additional indications for younger ages and expanded genetic variants. The current approval extends the indication to cystic fibrosis patients aged 2 years and older with any variant that shows response to the medication based on clinical data, in vitro data, or demonstrated production of CFTR protein.
Both medications carry a risk for liver injury, and patients should be assessed for liver function before starting the drugs and monitored routinely, according to the company. The medications are contraindicated for those with severe hepatic impairment and only recommended based on risk/benefit analysis in patients with moderate hepatic impairment.
Detailed prescribing information is available for Alyftrek and Trikafta.
The supporting research was funded by Vertex Pharmaceuticals.
