FDA Approves Triple Combination Therapy for Cystic Fibrosis

FEBRUARY 13, 2018
Kevin Kunzmann
cystic fibrosis, FDA, SYMDEKO, approval, drugThe US Food and Drug Administration (FDA) has approved a combination therapy for cystic fibrosis (CF) that could eventually serve as the foundation of triple-therapy regimens.

Combination tezacaftor/ivacaftor and ivacaftor (SYMDEKO) has been approved 2 weeks in advance of its PDUFA action date, for the treatment of CF in people aged 12 years and older who have 2 copies of F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, or who have at least 1 tezacaftor/ivacaftor-responsive mutation.

The therapy is the third Vertex Pharmaceuticals drug approved for patients with CF in the US, and is projected to serve as clinical backbone for next-generation therapies that could treat up to 90% of patients with its wider range of efficacy than previous combination drugs.

SYMDEKO’s approval was backed by the results of a couple of phase 3 studies (EVOLVE, EXPAND) which tested the combination therapy in nearly 750 patients with CF aged 12 years and older who have 2 copies of F508del mutation in the CFTR gene.

The larger EVOLVE study, a 24-week evaluation of 500-plus CF patients, found that the combination therapy provided clinically meaningful improvements in lung function and a reduction in pulmonary exacerbations, versus a placebo population.

In the EXPAND study, the tezacaftor/ivacaftor combination was compared to ivacaftor monotherapy or placebo treatment groups in approximately 250 CF patients evaluated over 8 weeks. Researchers noted improvements in lung function patients treated with tezacaftor/ivacaftor and ivacaftor monotherapy versus those treated with placebo.

CF currently effects about 75,000 people in North America, Europe, and Australia combined. The genetic disease causes abnormal mucus buildup that creates chronic infections and progressive damage. The median patient age of death is in the mid-to-late 20s.

The value of oral ivacaftor — marketed as KALYDECO in the US — is in its ability to keep CFTR proteins at the cell surface open for a longer period of time. This improves the transport of salt and water across the cell membrane, helping clear airways from mucus.

Following the approval on Monday, Vertex announced intentions to launch SYMDEKO this week. The tezacaftor/ivacaftor combination therapy’s Marketing Authorization Application was previously validated by the Euroepean Medicines Agency, and the company anticipates it will be approved for European markets in late 2018.

Jeffrey Leiden, MD, PhD, Vertex chairman, president, and chief executive officer, said the SYMDEKO approval offers many patients an important, third treatment option in CF.

“This approval is an important milestone in our journey to treat every person with CF, and we remain committed to urgently advancing our efforts to develop new medicines that treat the underlying cause of CF for the many people still waiting,” Leiden said.

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