FDA Approves Expanded Use of Orkambi in Young Cystic Fibrosis Patients

Orkambi pic
Orkambi
Image: investors.vrtx.com

A physician and surgeon focused on ear, nose, and throat issues, Dr. William B. Clark treats patients through ENT Associates of Northwest Florida, a practice he owns. Outside of his professional life, Dr. William B. Clark has provided support to various nonprofit organizations, including those working to fight cystic fibrosis.

An additional 2,400 children who have cystic fibrosis now have a new treatment option available to them. The US Food and Drug Administration (FDA) recently gave its approval for the expanded use of Orkambi, also known as lumacaftor/ivacaftor, for children age 6 to 11 who have two copies of gene mutation F508del. Factoring in this new cohort, nearly 11,000 children in the country are now eligible to be treated with the drug.

Vertex Pharmaceuticals is behind the drug’s development, which was aided by considerable funding and support from the Cystic Fibrosis Foundation. The company is currently in the third phase of a clinical trial that it hopes will allow for the use of the drug by patients from 2 to 5 years old.