Vertex Receives FDA Approval for Continuously Manufactured Drug Product

FDA approves Vertex's cystic fibrosis drug, Symdeko (tezacaftor/ivacaftor and ivacaftor), which is made using a continuous manufacturing process.

FDA approved the Vertex cystic fibrosis drug, Symdeko (tezacaftor/ivacaftor and ivacaftor), for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor, the company announced in a Feb. 12, 2018 press release. The drug is Vertex's third medicine approved to treat the underlying cause of CF. Vertex is ready to launch Symdeko and will begin shipping it to pharmacies in the United States, the company announced.

"We've already seen the 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," said Patrick Flume, MD, director of the Medical University of South Carolina Cystic Fibrosis Center and Principal Investigator for one of the drug’s Phase III studies, in the press release. "In particular, Symdeko is an important treatment option for patients who either never started or discontinued Orkambi [lumacaftor/ivacaftor], and it also provides increased benefit over Kalydeco [ivacaftor] alone for patients with residual function mutations."

The European Medicines Agency has validated the Marketing Authorization Application (MAA) for the tezacaftor/ivacaftor combination. Vertex expects approval in the EU in the second half of 2018, the company reported in the press release.

Symdeko is Vertex’s second approved drug made using a continuous manufacturing process. Orkambi, a cystic fibrosis drug approved by FDA in July 2015, was the first ever oral solid dosage drug approved to be produced using a continuous manufacturing process.

Source: Vertex