The U.S. FDA approved a new cystic fibrosis medication that is expected to provide treatment for 90% of patients with this debilitating and life-shortening condition. Late Monday, the FDA greenlit Vertex Pharmaceuticals’ Trikafta.
Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) was approved for use in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gene, which is the most common CF-causing mutation. Trikafta helps the CFTR protein made by the CFTR gene mutation function more effectively.
With the approval, Vertex said that for the first time, approximately 6,000 patients with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease. Not only that, but 12,000 people with one or two F508del mutations who are currently eligible for one of Vertex’s three other FDA-approved cystic fibrosis medicines are now also eligible for Trikafta, the company said.
Cystic fibrosis is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. There are approximately 2,000 known mutations in the CFTR gene. The absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.
Trikafta earned approval bases on two successful Phase III trials in CF patients ages 12 and up who had at least one F508del mutation and one minimal function mutation and in people with two F508del mutations. In both trials, Trikafta increased the percent predicted forced expiratory volume in one second (ppFEV1). Approval for the drug came about five months ahead of its PDUFA date, which was originally set for March 19, 2020. When the New Drug Application for Trikafta was accepted in August, the regulatory agency granted Priority Review to the drug, Vertex said.
Regarding that quick approval, acting FDA Commissioner Ned Sharpless said the quick turnaround in the approval process is due to the agency’s commitment to speeding up approval for complex disease treatments, while still maintaining the rigorous standards for review.
“In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients’ quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options. That’s why we used all available programs, including Priority Review, Fast Track, Breakthrough Therapy, and orphan drug designation, to help advance today’s approval in the most efficient manner possible, while also adhering to our high standards,” Sharpless said in a statement.
Jeffrey Leiden, chairman and chief executive officer of Vertex, called the approval of Trikafta a “milestone for CF patients and their families,” as well as for Vertex itself. He said the approval of Trikafta capped off a 20-year journey to develop a breakthrough treatment for the majority of cystic fibrosis patients.
“For approximately 6,000 people with CF in the U.S., Trikafta is the first medicine that can treat the underlying cause of their disease,” Leiden said in a statement. “I want to personally thank the hundreds of Vertex scientists who have been working on this program for nearly 20 years – many of whom have dedicated their entire careers to changing the course of this disease; the CF Foundation which has provided support, encouragement and help throughout the journey; and most importantly the thousands of patients, caregivers, doctors and advocates who have courageously and persistently worked side-by-side with us to get to where we are today.”
Vertex is seeking approval of Trikafta in Europe and is also evaluating the medication in CF patients aged 6 to 11 with F/MF and F/F CF mutations in an ongoing Phase III study. The company said it is planning future studies for patients under the age of six years old.