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Vertex Orkambi's Label Expanded in U.S., Outlook Revised

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Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that its cystic fibrosis (CF) drug, Orkambi, was approved in the U.S. for the treatment of CF in patients aged 6–11 years who have two copies of the F508del mutation. The company said that the drug will be available to the eligible population shortly.

Vertex also plans to file a Marketing Authorization Application (MAA) variation in the EU for the same indication in the first half of 2017.

Note that Orkambi is a combination of lumacaftor and Kalydeco (ivacaftor), a marketed CF drug in the company’s portfolio. Orkambi was approved in the U.S. and the EU in Jul 2015 and Nov 2015, respectively, for the treatment of CF in patients aged 12 years and older, who have two copies of the F508del mutation in their CF transmembrane conductance regulator (CFTR) gene. In the first six months of 2016, Orkambi raked in sales of $468.7 million.

Meanwhile, Vertex has lowered its 2016 guidance for Orkambi sales to the range of $950–$990 million from the previously expected $1.0–$1.1 billion. The revision was primarily due to slower-than-anticipated launch of Orkambi in Germany and less-than-expected refills in the months of July and August. Sales of the drug are expected in the range of $230–$235 million in the third quarter of 2016.

CF, a rare, life-threatening disease, is estimated to affect approximately 75,000 people in the U.S., Europe and Australia. The company expects the latest label expansion of Orkambi to cover approximately 11,000 patients in the U.S.

Although Vertex is the first company to successfully develop drugs for the treatment of the underlying cause of CF, several other players in the pharma and biotech space are involved in the development of drugs for this indication. Nivalis Therapeutics, Inc. is currently evaluating its own CF candidate, N91115, in a phase II study. Top-line results of the study are expected by the end of 2016.

Earlier this year, Galapagos NV (GLPG - Free Report) and AbbVie Inc. (ABBV - Free Report) expanded their collaboration agreement for CF and aimed to develop a triple CFTR combination therapy.

We expect investor focus to remain on further news related to the CF market.

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