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.

Precision Medicine Offers New Hope for People with Cystic Fibrosis

Cystic Fibrosis Foundation pic
Cystic Fibrosis Foundation
Image: cff.org

Dr. William B. Clark concentrates his medical practice on surgery of the head and neck at ENT Associates of Northwest Florida. Outside of his practice, Dr. William B. Clark donates to charities such as the Cystic Fibrosis Foundation.

A genetic disease, cystic fibrosis (CF) causes lung infections that hinder breathing. It creates a thick mucus that affects the pancreas as it tries to absorb essential nutrients and the lungs as they try to supply the body with oxygen.

Having CF in her early 30s, longer than thought possible, Emily Kramer-Golinkoff has stepped forward as an advocate for the foundation. Telling her story in many media appearances, she was honored as a Champion of Change by President Obama for raising some $1.5 million for CF research.

Emily Kramer-Golinkoff has been pushing for a philosophy of care known as precision medicine. This innovative concept emphasizes treatment of patients as they are: individuals with unique lifestyles, environments, and genetic traits. It enables medical care providers to better comprehend the complexities of each patient’s circumstances. The possibilities it holds for creating better, targeted treatments are just beginning to be realized.