Indications Expanded for Three Cystic Fibrosis Drugs

December 21, 2020 – The U.S. FDA has approved expanded indications for three medications from Vertex Pharmaceuticals that are indicated to treat cystic fibrosis (CF), with patients selected based on the presence of certain genetic mutations tied to CF. The medications are Trikafta® (elexacaftor/ tezacaftor/ivacaftor, ivacaftor), Symdeko® (tezacaftor/ivacaftor, ivacaftor), and Kalydeco® (ivacaftor).

CF is a rare genetic disorder that can affect multiple organs in the body, including the lungs, digestive tract, and liver. It occurs when mutations in the CFTR gene lead to defective or absence CFTR protein, which is necessary to manage the movement of salt and water into and out of the body’s cells. This causes an abnormally thick, sticky mucus to form in areas such as the lungs, and ultimately shortens the patient’s lifespan. The average lifespan for patients with CF is 44 years, with death most often occurring due to respiratory problems. Trikafta, Symdeko, and Kalydeco work by helping to improve the balance of salt and water inside and outside of the body’s cells.

Under the newly expanded indications, Trikafta has been approved to treat 177 additional mutations tied to CF, while Symdeko is now approved for 127 additional mutations, and Kalydeco has been approved for an additional 59 mutations. Vertex Pharmaceuticals anticipates these expansions will allow more than 600 additional patients diagnosed with CF to be eligible for treatment with Trikafta, Symdeko, or Kalydeco.

Each medication is taken daily, by mouth, with the recommended dosing for Symdeko and Kalydeco dependent on individual patient factors such as weight and age. All three drugs may require dose modifications based on whether the patient has hepatic impairment and/or is taking other medications. Trikafta is indicated for use in patients age 12 and up only, while Symdeko and Kalydeco are approved for use in patients as young as four months.