New label extensions for Alyftrek and Trikafta make approximately 95% of the cystic fibrosis population in the United States eligible for treatment.
RT’s Three Key Takeaways:
- Expanded Eligibility: The FDA approved label extensions for Alyftrek and Trikafta, making approximately 95% of people with cystic fibrosis in the US eligible for these therapies.
- Broad Variant Coverage: The new indications include any cystic fibrosis transmembrane conductance regulator (CFTR) gene variant that results in protein production, adding approximately 800 people to the eligible patient pool.
- Clinical Support: Approval was based on clinical and in vitro data demonstrating response across more than 500 genetic variants for each medication.
The FDA has approved expanded indications for two cystic fibrosis (CF) treatments, Alyftrek and (vanzacaftor/tezacaftor/deutivacaftor) and Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), according to Vertex Pharmaceuticals.
This expansion allows approximately 95% of all people with CF in the US to be eligible for treatment with a cystic fibrosis transmembrane conductance regulator (CFTR) modulator.
The FDA approved Alyftrek for patients aged 6 years and older who have a variant in the CFTR gene that is responsive based on clinical or in vitro data, or that results in the production of CFTR protein. The agency also expanded the indication statement for Trikafta for patients aged 2 years and older. These changes mean approximately 800 additional people with a clinical diagnosis of CF are now eligible for a medication that treats the underlying cause of the disease for the first time.
“This groundbreaking approval represents more than 20 years of innovation in CF, including testing over 600 variants in our laboratory, demonstrating clinical effects in large clinical trials, and studying younger people with CF so they can be treated as early as possible,” said Carmen Bozic, MD, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, in a news release.
The label expansion was supported by data from 564 variants demonstrating response to Alyftrek and 521 variants demonstrating response to Trikafta. Bozic noted that any variant resulting in the production of CFTR protein is now included in the labels, validating that the medicines can restore function regardless of the location of the variant in the CFTR protein.
