Vertex Pharmaceuticals Incorporated (VRTX) provided a comprehensive overview of its initiatives geared towards the development, approval and launch of new medications for the treatment of patients suffering from cystic fibrosis (CF). The company also provided its financial guidance for 2015.
Kalydeco Label Expansion to Continue in 2015
Vertex Pharma’s lead product, Kalydeco, is currently approved for the treatment of patients (six years and above) with CF who have specific genetic mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene. With the label expanding through 2014, Kalydeco can be used in the U.S. in CF patients aged six years and older with the R117H mutation or any one of these 8 mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D.
Vertex Pharma continues to work on expanding Kalydeco’s label and is currently seeking FDA approval for the treatment of children aged 2-5 years who have the G551D or one of the 8 additional gating mutations – a response is expected by Mar 17, 2015. In addition to this, Kalydeco is under review in the EU for the same patient population as well as for patients aged 18 years and older with the R117H mutation.
According to the company, more than 3,100 patients are currently eligible for treatment with Kalydeco and this number is expected to go up to more than 3,700 by the end of 2015.
Apart from Kalydeco, Vertex has a couple of promising CF correctors in its pipeline – lumacaftor and VX-661. Both are being evaluated in combination with Kalydeco.
Kalydeco – Lumacaftor Combination Under FDA Priority Review
Vertex Pharma informed that the FDA has granted priority review to the company’s new drug application for Kalydeco in combination with lumacaftor for use in CF patients (age ≥12 years) who have two copies of the F508del mutation in the CFTR gene. A response from the FDA is expected by Jul 5, 2015. The combination is also under review in the EU. Approval would provide the company with access to about 22,000 people in North America, Europe and Australia including 8,500 in the U.S. and 12,000 in Europe.
The company plans to commence a study on this combination in children aged 6-11 years who have two copies of the F508del mutation in the first half of 2015.
Kalydeco – VX-661 Combination to Enter Phase III Study
The company plans to initiate a phase III pivotal program, consisting of four studies, to evaluate the efficacy and safety of VX-661 in combination with Kalydeco in February this year, pending regulatory discussions and data (expected in the first quarter of 2015) from an ongoing phase II study.
Kalydeco – Key Growth Driver in 2015
Vertex Pharma provided its preliminary outlook for 2015. The company is expecting 2015 Kalydeco revenues in the range of $560 million to $580 million, up from approximately $460 million in 2014.
Vertex Pharma expects combined R&D and SG&A expenses for 2015 in the range of $1.05 billion to $1.1 billion. The increase reflects launch preparation activities for the Kalydeco-lumacaftor combination and the planned pivotal phase III program for the Kalydeco-VX-661 combination.
The company expects fourth quarter 2014 Kalydeco revenues of around $120 million. Vertex Pharma will be reporting fourth quarter results on Jan 28, 2015.
Vertex Pharma currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Alexion Pharmaceuticals, Inc. (ALXN), AMAG Pharmaceuticals, Inc. (AMAG) and Amgen Inc. (AMGN). All three carry a Zacks Rank #1 (Strong Buy).
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