Vertex's Third Cystic Fibrosis Treatment Gets FDA Approval

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Vertex Pharmaceuticals Incorporated (VRTX Free Report) announced that the FDA has granted approval to its cystic fibrosis (CF) corrector, tezacaftor (VX-661), in combination with ivacaftor (Kalydeco) under the brand name, Symdekotm. The combo therapy is approved to treat the underlying cause of CF in patients aged 12 years and above with certain mutations in the CF transmembrane conductance regulator gene.

This approval marks the company’s third treatment for the underlying cause of CF. The company said that it is ready to launch the product in the U.S. market this week. With this nod from the regulatory agency, Vertex can address a significantly larger CF patient population in the United States.

Notably, the combination regime is also under review in the EU with a decision expected in the second half of 2018.

Vertex’s share price has soared 73.2% in a year’s time, comparing favorably with the industry’s decrease of 2.5%.

The aforementioned approval was supported by positive data from two phase III studies on tezacaftor/ivacaftor combination, EVOLVE and EXPAND. Both trials met primary endpoints and demonstrated statistically significant improvements in lung function in two different CF-patient population. The combination treatment was found to be well tolerated in both cases.

The company presently markets two CF drugs, Kalydeco and Orkambi, and also boasts a strong CF pipeline. Its broad portfolio of next-generation CF correctors could bring in multi-billion dollar sales for the company, if approved.

Notably, Vertex is evaluating some next-generation CFTR correctors (VX-152, VX-440, VX-659 and VX-445) as part of a triple combination with tezacaftor and ivacaftor.

Data from VX-152 and VX-440 phase II and VX-659 phase I triple combination programs presented in July 2017 showed that all three combos led to a marked improvement in lung function. The company is also developing the fourth next-generation corrector, VX-445, in a phase II study with data expected in early 2018.

Meanwhile, the company is looking to buy CF candidates, which can be put together with tezacaftor and ivacaftor to create triple combinations. Last July, Vertex bought Concert Pharmaceuticals, Inc.’s (CNCE Free Report) CF pipeline candidate, VX-561 (CTP-656). The company is developing the candidate as a promising once-daily triple combination compiled with other pipeline drugs to treat the underlying cause of CF.

We remind investors that the CF market represents huge commercial potential. It is a rare, life-threatening disease estimated to affect about 75,000 people in North America, Europe and Australia. The CF combination regimens, if approved, can further strengthen the market position, translating into higher revenues.

Zacks Rank & Key Picks

Vertex carries a Zacks Rank #3 (Hold). Two better-ranked stocks in the health care sector are XOMA Corporation (XOMA Free Report) and Exelixis, Inc. (EXEL Free Report) . While XOMA sports a Zacks Rank #1 (Strong Buy), Exelixis carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

XOMA’s loss per share estimates have narrowed from 99 cents to 42 cents for 2018 over the last 60 days. The company came up with an average beat of 47.92%. The stock has skyrocketed 430% in the last 12 months.

Exelixis’ earnings per share estimates have been revised upward from 72 cents to 77 cents for 2018 over the last 60 days. The company pulled off a positive surprise in all the trailing four quarters with an average beat of 572.92%. Share price of the company has rallied 36.4% in a year’s time.

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