FDA OKs Combo Treatment for Cystic Fibrosis Caused by Certain Mutations

FEBRUARY 13, 2018
FDA officials have approved tezacaftor/ivacaftor and ivacaftor (Symdeko, Vertex Pharmaceuticals) for the treatment of cystic fibrosis (CF) in individuals aged 12 years and older, according to the pharmaceutical company’s press release. The drug is indicated for those with 2 copies of the F508del mutation or one mutation that is responsive to the treatment.

The approval is based on the results of two phase 3 studies, EVOLVE and EXPAND, that included approximately 750 people with CF aged 12 years and older with 2 copies of the F508del mutation or with one F508 and one mutation that results in residual CFTR function. Patients treated with tezacaftor/ivacaftor and ivacaftor demonstrated statistically significant and clinically meaningful improvement in lung function and other measures of disease.

The tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface where ivacaftor can increase the amount of time the protein stays open.

“We’ve already seen a significant impact that disease-modifying medicines can have on patients and are incredibly pleased that there is now a third treatment option that enables more patients to benefit from CFTR modulation,” Patrick Flume, MD, Director of the Medical University of South Carolina Cystic Fibrosis Center and Principal Investigator for the EXTEND study, said in a press release. “In particular, Symdeko is an important treatment option for patients who either never started or discontinued Orkambi, and it also provides increased benefit over Kalydeco alone for patients with residual function mutations.”

The most common adverse effects associated with tezacaftor/ivacaftor and ivacaftor include headache, nausea, sinus congestion, and dizziness.

The European Medicines Agency has validated the Marketing Authorization Application for tezacaftor/ivacaftor combination and the company expects approval in the EU in the second half of 2018.

FDA Approves SYMDEKO (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene [news release]. Vertex’s website. Accessed February 13, 2018.