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Symdeko was approved for patients age 12 or older who are homozygous for the F508 deletion of the CFTR gene, or who have another mutation that responds to Symdeko.
The drug should be available this week in the U.S, the company reported.
Approval was largely based on the EVOLVE and the EXPAND studies, phase 3 industry-sponsored randomized trials published last November in the New England Journal of Medicine. Symdeko-treated patients had improvements in lung function and other clinical outcomes, with a favorable safety profile. The enrolled patients all had one or two deletions of F508.
Vertex Pharmaceuticals is expected to charge insurance companies and the government about $292,000 per patient for a yearly supply of Symdeko, according to industry analysts.
That's about 7% higher than an existing Vertex drug combination product sold as Orkambi (ivacaftor and lumacaftor), which payers are already covering. In clinical trials, Symdeko was better tolerated and more effective than Orkambi, according to analysts. Vertex's stated longer term goal is a three-drug regimen that could treat almost all the genetic mutations responsible for cystic fibrosis.
Unlike Orkambi, Symdeko's label doesn't contain a warning about respiratory events or blood pressure problems. These safety and convenience concerns may have limited use of Orkambi. Symdeko is believed to be potentially twice as effective as Orkambi in treating cystic fibrosis.
In SEC filings, Vertex reportedly announced anticipating $2.8 billion in annual revenues from its three cystic fibrosis drugs (Kalydeco, Symdeko and Orkambi), an increase of $150 million over the previous year.
Vertex is also reportedly working with Crispr Therapeutics on a cure for cystic fibrosis and several other genetic conditions using gene editing. According to news reports, the two companies signaled intent to start trials on gene editing treatment or cures of sickle cell disease and beta thalassemia in 2018.
Sources: MedPage Today, Investors Business Daily, Reuters