In a significant breakthrough for cystic fibrosis therapeutics focusing on child-care, Vertex Pharmaceuticals’s TRIKAFTA, has received US FDA approval.
Post the US Food and Drug Administration of Vertex’s TRIKAFTA; the drug may now be used to treat cystic fibrosis (CF) in children between the age group, six-eleven (6-11) years.
The drug is designed to treat conditions of F508del mutation. The mutation in CF transmembrane conductor regulator (CFTR) is a significant medical gap, and the latest approval is expected to address this challenge.
TRIKAFTA is not a new drug and was earlier recommended for patients of at least 12 years of age and above. US, Switzerland, Israel, Australia have already been using TRIKAFTA for quite some time.
Conditions such as F508del mutation or in vitro responsive mutation have been treated with TRIKAFTA.
Following the recent approval of TRIKAFTA for cystic fibrosis, the drug would henceforth be available in various power such ivacaftor 75 mg, ivacaftor 75 mg, and tezacaftor 25 mg.
Responding to this vital milestone, President and CEO, Vertex Pharmaceuticals Inc. said, “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,.”
Adding further, she said, “We can now reach approximately 1,500 newly eligible children in the US, and we continue to pursue approval for this expanded indication in other countries.”
Prior to TRIKAFTAR approval, Vertex carried out a six-month clinical study of the Phase 3 level. In all, 66 participants were taken between the age of six and eleven years.