VRTX Vertex Pharmaceuticals Inc

Vertex Pharmaceuticals Inc. (VRTX) received encouraging news when the U.S. Food and Drug Administration (FDA) approved Kalydeco for an additional indication. Kalydeco was approved for treating patients (age ≥ 6 years) suffering from cystic fibrosis (CF) who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Kalydeco is currently marketed for treating CF patients (age ≥ 6 years) who have at least one copy of the G551D mutation. Following the approval for the new indication, Kalydeco can be used to treat CF patients with the following nine mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

Kalydeco’s approval for the new indication was based on phase III data that demonstrated statistically significant improvements in lung function in patients on Kalydeco monotherapy. The safety profile was similar to that seen in earlier phase III studies. Approval in the U.S. for the new indication was sought in Oct 2013. Similar submissions for Kalydeco were also made in Europe, Canada and Australia for the same indication.

Vertex Pharma is also evaluating Kalydeco in children with CF in the age group of 2–5 years who have a gating mutation. The study is fully enrolled and currently ongoing. The company expects data from the study in the second quarter of 2014. The company intends to seek U.S. approval in this indication in the second half of 2014. However, in December last year, Vertex Pharma suffered a setback when its phase III study on Kalydeco for the indication of cystic fibrosis (CF) in patients (age ≥ 6 years) with R117H mutation, failed to meet its primary endpoint. The company is also evaluating Kalydeco for other indications.

Meanwhile, Vertex Pharma has been witnessing encouraging Kalydeco sales since its launch. The company reported Kalydeco sales of $371.3 million in the year 2013, an increase of 116.4% from the year-ago period. Kalydeco sales are expected in the range of $470 million to $500 million in 2014.

Vertex Pharma maintained its total revenue guidance for 2014 in the range of $570 million to $600 million. The Zacks Consensus Estimate of $603 million is slightly above the company’s guidance range.

Vertex Pharma carries a Zacks Rank #3 (Hold). Some better-ranked stocks include Alexion Pharmaceuticals, Inc. (ALXN), Actelion Ltd. (ALIOF) and Alkermes (ALKS). While Alexion holds a Zacks Rank #1 (Strong Buy), Actelion and Alkermes carry a Zacks Rank #2 (Buy).
 

 
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