New Cystic Fibrosis Medication Treats 90% of Patients, Priced at $300,000/Annually
On Monday October 21st, the U.S. Food and Drug Administration (FDA) announced the approval of Trikafta®, the first triple combination therapy, for cystic fibrosis (CF). Trikafta® is approved for patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the CF population. Vertex Pharmaceuticals, the manufacturer of Trikafta®, announced it would cost about $311,000 annually.
Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. CF occurs as a result of CFTR gene mutation which causes the CFTR protein to become dysfunctional. When this protein does not work effectively it results in mucus build up in lungs, pancreas and liver preventing them from working appropriately. Currently, the life expectancy for patients diagnosed with CF is about 38 years but has been increasing steady over the last 10 years with the approval of new medications. It is estimated approximately 30,000 individuals in the U.S. are living with CF.
Trikafta® will join three other CFTR modulator treatments. CFTR modulator therapies are designed to correct the malfunctioning protein made by the CFTR gene. All three alternatives are similarly priced to Trikafta® (Symdeko® $292,000, Kalydeko® $307,000, and Orkambi® at $273,000).