FDA Approves Vertex’s Symdeko to Treat CF Patients 12 and Older

FDA Approves Vertex’s Symdeko to Treat CF Patients 12 and Older

The U.S. Food and Drug Administration (FDA) has approved the third targeted therapy developed by Vertex Pharmaceuticals for patients with cystic fibrosis (CF)Symdeko can now be used in the United States to treat CF patients ages 12 and older who have two copies of the F508del mutation in the CFTR gene, or have at least one mutation that is responsive to the drug’s components.

Symdeko is the combination of tezacaftor plus ivacaftor (sold under the brand name Kalydeco). It takes advantage of tezacaftor’s ability to restore the CFTR protein’s natural form, and of the CFTR activity enhancer potential of ivacaftor to achieve improved outcome for cystic fibrosis patients.

Vertex will start distributing Symdeko this week to pharmacies across the United States.

“The approval of Symdeko, our third disease-modifying CF medicine, offers many patients an important new treatment option,” Jeffrey Leiden, MD and PhD, chairman, president, and CEO of Vertex, said in a press release.

“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.

The European Medicines Agency (EMA) is currently reviewing Vertex’s Marketing Authorization Application (MAA) for Symdeko, and a final decision by the EMA is expected in the second half of 2018.

Both the FDA and EMA regulatory submissions were supported by clinical data compiled in the EVOLVE (NCT02347657) and EXPAND (NCT02392234) Phase 3 trials.

These studies included about 750 CF patients who were treated with Symdeko or placebo. Symdeko treatment was shown to significantly improve lung function and other health measures compared to placebo, with a favorable safety profile. The positive effects of the treatment were found to be sustained for up to 48 weeks, as reported in the rollover EXTEND (NCT02565914) trial.

“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 of the EXTEND study.

“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 alone for patients with residual function mutations,” Flume added.

To facilitate access to the newly approved CF therapy, the Vertex GPS – guidance and patient support program – has a team available to help eligible patients understand their insurance benefits. The program also offers a co-pay assistance program for patients that meet specific criteria but can’t afford the therapy. For more information, visit www.VertexGPS.com.

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