Vertex's Orkambi Wins EU Nod to Treat Cystic Fibrosis in Kids

Vertex Pharmaceuticals Incorporated VRTX announced that the European Commission (EC) has granted an approval to a regulatory application seeking Orkambi’s label expansion for its use in children aged two to five years with cystic fibrosis (CF), who have two copies of the F508del mutation.

The EU approval was based on data from a phase III open-label safety study evaluating 60 patients, who showed good tolerance for a 24-week regimen under the influence of Orkambi and the safety profile was similar to that of patients aged six years or older.

Following this nod, Orkambi becomes the first and only approved medicine in Europe to treat CF in kids aged from two to five years. Approximately 1,500 children in Europe within the mentioned age group suffer this most common form of CF disease, thus making Orkambi eligible to treat an expanded patient population.

Orkambi is already approved in the United States and Europe for treating CF in patients aged six or above who have two copies of the F508del mutation. The drug was given a nod in the United States for the two to five-year age group last August.

Shares of Vertex were up 2.19% on Monday following this news. In fact, the stock has rallied 20.2% in the past year against the industry’s decline of 21.4%.

However, Orkambi sales have been waning of late due to patients’ switch to Vertex’s third and the newest CF medicine — Symdeko — from Orkambi, which targets the same patient population. In the first nine months of 2018, Orkambi generated sales of $947 million, down 0.8% year over year.

Notably, CF is Vertex’s main area of focus. Apart from Orkambi, its other two approved CF products are Kalydeco and Symdeko, which are constant revenue drivers. Total CF product revenues were $2.2 billion in the first nine months of 2018, reflecting a 46.7% surge year over year.

Evidently, Vertex’s CF pipeline is also quite strong. The company is evaluating two next-generation CFTR correctors (VX-659 and VX-445) in phase III studies as part of a triple combination with tezacaftor and ivacaftor.

Studies on Vertex’s triple combination CF regimens are advancing fast. The CF triple-pill regimes are crucial for long-term growth as these have potential to treat up to 90% of CF patient population.

Zacks Rank & Stocks to Consider

Vertex currently carries a Zacks Rank #4 (Sell).

Better-ranked stocks in the healthcare sector include Genmab A/S GNMSF, Acorda Therapeutics, Inc. ACOR and Aduro Biotech, Inc. ADRO, all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Genmab’s earnings estimates have moved 4% north for 2019 over the past 60 days.

Acorda’s loss per share estimates have been narrowed 1.3% for 2019 in the last 60 days.

Aduro’s loss per share estimates have been narrowed 4.5% for 2019 over the past 60 days.

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