Clinical Vertex scores FDA nod for kids' cystic fibrosis drug...

Vertex scores FDA nod for kids’ cystic fibrosis drug Trikafta


Trikafta was approved by FDA for the treatment of Cystic Fibrosis in patients aged 6 to 11 years, after receiving approval to be used in patients of ages 12 and up.

The FDA has authorized Vertex Pharmaceuticals to manufacture Trikafta for the treatment of Cystic Fibrosis in patients of ages 6 to 11 years.

This authorization is only for the children who have at least one F508del mutation in CFTR (Cystic Fibrosis Transmembrane Conductance) gene or a CFTR gene mutation that reacts to the treatment depending on its in-vitro studies.

Trikafta was approved by FDA in 2019 for the treatment of cystic fibrosis patients of ages 12 and up. It has also received authorization in countries such as Australia, Europe, and United Kingdom.

The drug is a combination of three APIs, elaxacaftor, ivacaftor, and tezacaftor. According to the manufacturer, the medication is now available in a new dose as a result of the approval.

Vertex Pharmaceuticals CEO and president Reshma Kewalramani 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.”
In phase 3, open-label, multi-center clinical trial, the firm evaluated the effectiveness, safety, and pharmacokinetic properties of the drug in 66 children of ages 6 to 11 years, for 24 weeks.
Two F508del mutant variants or one F508del mutant variant and one reduced functionality mutation were present in the patients.

The data obtained from the previous drug studies in individuals of ages 12 and up have shown similar safety data and well-tolerated treatment schedules for Trikafta.

The detailed findings of phase 3 clinical trial were published in the American Journal of Respiratory and Critical Care Medicine by Vertex Pharmaceuticals.

The legislative approval of Trikafta as Kaftrio is being sought by the company in the United Kingdom and Europe for the treatment of cystic fibrosis in patients of ages 6 to 11 years.

This year, the company will also seek approval in Australia, Switzerland, and Israel.

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