Vertex's Cystic Fibrosis Drug Given The Okay For Pediatric Use In Europe

Vertex Pharmaceuticals, Inc.VRTX announced that the European Commission has approved an extension of the marketing application for its cystic fibrosis product, Orkambi. With the approval, Orkambi's marketing label in EU will now include use in children with CF aged between six and 11 years and have two copies of the F508del mutation.

The label expansion approval in EU was based on positive data from asix-month phase III efficacy study in this patient population. Data from the study, presented in November 2016, demonstrated that treatment with Orkambi led to a statistically significant improvement in absolute change in lung clearance index – a measure of lung function in early CF disease – compared with placebo through 24 weeks of treatment.

Approval for this age group would expand the targeted patient population by about 3,400 patients.

In Ireland, Vertex already has reimbursement agreements for use in these children while in other countries, a country-by-country reimbursement process will now be initiated by Vertex.

Please note that in the United States, Orkambi is already marketed for this patient population since September 2016.

Year 2017 was a tremendous one for Vertex with positive data read-outs and regulatory approvals leading to an increase in eligible patient population for Kalydeco & Orkambi. With a consistent expansion in patient population, Vertex's CF product revenues increased through 2017. With the latest label expansion approval in EU, it looks like the trend will continue in 2018.

The company is optimistic that the positive trend in CF sales will continue in 2018 as it gains additional reimbursement approvals for Orkambi in ex-U.S. markets and gets approval to launch tezacaftor (VX-661)/ivacaftor combination medicine.

The stock has surged 92.1% in the past year, substantially outperforming the 1% increase registered by the industry.

Estimates have also moved northward in the past 30 days.

Vertex's CF pipeline is also quite strong with a broad portfolio of next-generation CF correctors. Vertex also made decent clinical progress across multiple CF studies in 2017 and has meaningful pipeline catalysts. Investor focus is on triple combination CF regimens, which are crucial for long-term growth as these have the potential to treat up to 90% of CF patients. Vertex plans to initiatepivotal phase III studies on up to two of the four triple combination regimens in the first half of 2018.

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