Trikafta From Vertex Pharmaceuticals Approved By US FDA As New Treatment Drug For Cystic Fibrosis
(elexacaftor/ivacaftor/tezacaftor) from Vertex Pharmaceuticals
is the first triple combination therapy to be approved by the US FDA and made available to treat patients with the most common cystic fibrosis
mutation. Its list price is US$350,000 a year, same as one of the maker's earlier treatments for the genetic disease.
has been approved for patients aged 12 and older who have at least one F508del in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene. This includes about 90% of cystic fibrosis,
or about 28,000 people in the United States, according to the FDA. Globally the rough number of individuals inflicted with this disease that have the gene stands at about 1.8 million individuals.
Altogether there are about 2,000 known mutations of the CFTR gene, but the F508del mutation is the most common. Cystic fibrosis i
s the result of a defective protein caused by the gene mutation.
Current drugs that target the defective protein can be used to treat some patients, but many patients have mutations that don't respond to those treatments, the FDA said.
Trikafta's approval is based on results of two clinical trials involving 510 patients.The trials assessed a measure of lung function called percent predicted forced expiratory volume in one second (ppFEV1). It measures how much air a person can exhale during a forced breath.
In the first trial, average ppFEV1 among patients who took Trikafta improved 13.8% compared to those who took a placebo. In the second trial, the drug increased average ppFEV1 by 10% compared to the drugs tezacaftor/ivacaftor.
Acting FDA Commissioner Dr. Ned Sharpless said the approval was an outgrowth of efforts to speed development of new therapies for complex diseases.In an interview with Thailand Medical
News, he said, "Trikafta
's approval makes a new treatment available to most cystic fibrosis
patients, including adolescents, who previously had no options and giving others in the cys
tic fibrosis community access to an additional effective therapy."
is a progressive, life-threatening disease. It causes formation of thick mucus that builds up in the lungs,digestive tract and other parts of the body, resulting in severe respiratory and digestive problems and other complications, such as infections and diabetes.
The prescribing information for Trikafta
includes warnings related to elevated liver function tests (transaminases and bilirubin), use at the same time with other products that are inducers or inhibitors of another liver enzyme called Cytochrome P450 3A4 (CYP3A), and the risk of cataracts. Patients and their caregivers should speak with a health care professional about these risks and any medicines they take before starting treatment.