Regulatory Affairs

Vertex’s Symdeko cystic fibrosis drug secures FDA approval

PBR Staff Writer Published 13 February 2018

Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor) has been approved in the US to treat the underlying cause of cystic fibrosis (CF) in people aged 12 and over.

The approval from the US Food and Drug Administration (FDA) is for CF patients having two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or those having at least one mutation that is responsive to tezacaftor/ivacaftor.

Symdeko is a combination of 100mg/150mg of tezacaftor/ivacaftor and 150mg ivacaftor.

The drug’s approval for the life-shortening lung disorder has been driven from the findings of two phase 3 studies, EVOLVE and EXPAND.

In both the trials, patients subjected to Symdeko had shown statistically significant and clinically meaningful improvements in their lung function and other measures of disease.

The two late-stage trials featured around 750 CF patients, aged 12 and over, and who have two copies of the F508del mutation or with one F508del mutation and one mutation resulting in residual CFTR function.

Vertex chairman, president and CEO Jeffrey Leiden said: “The approval of Symdeko, our third disease-modifying CF medicine, offers many patients an important new treatment option.

“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.”

Vertex said it will start shipping it to pharmacies in the US this week.

In Europe, the tezacaftor/ivacaftor combination is being reviewed by the European Medicines Agency. Vertex anticipates an approval in the European Union in the second half of this year.

Earlier this month, Vertex said that VX-659 and VX-445, its two next-generation correctors will advance into phase 3 development as part of two different triple combination regimens for people with CF.


Image: Symdekotm is a combination of tezacaftor and ivacaftor. Photo: courtesy of Business Wire.