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Vertex's (VRTX) Symdeko Gets FDA Nod for Use in Children

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Vertex Pharmaceuticals Incorporated VRTX announced that the FDA has granted approval to Symdeko to treat eligible cystic fibrosis patients as young as six years of age.

With the latest approval, Symdeko can be prescribed to children with cystic fibrosis in the age group of 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Symdeko. The drug, which was until now approved to treat patients 12 years and above, is now approved for eligible CF patients 6 years of age and older.

This year so far, Vertex’ shares have rallied 11% compared with the industry’s increase of 6.4%.

Symdeko, a combination of Vertex’s other CF medicine, Kalydeco (ivacaftor) and tezacaftor/ivacaftor, was Vertex’s third CF medicine to get approved in the United States in February 2018 and in the European Union in November 2018 (marketed as Symkevi). Other than Symdeko and Kalydeco, Vertex markets another CF medicine, Orkambi (lumacaftor) in combination with ivacaftor.

Symdeko, in a very short time, has become the primary driver of CF revenues. It generated sales of $320.3 million in the reported quarter, reflecting an increase of 8.8% sequentially. Symdeko’s uptake is being driven by new patient starts as well as shift from Orkambi and Kalydeco.

Following the recent approval, once Symdeko is administered to the younger patients in the United States, it should further boost revenues from the drug.

Also, along with the approval in children, the FDA also granted approval to an additional dosage (tezacaftor 50 mg/ivacaftor 75 mg and ivacaftor 75 mg) of Symdeko tablets.

Meanwhile, Vertex plans to submit an application seeking approval for use in children aged 6 through 11 years in Europe in the second half of 2019.

Vertex’s three CF medicines are collectively approved to treat around 50% of the 75,000 CF patients in North America, Europe and Australia. Meanwhile, Vertex evaluated two next-generation CFTR correctors (VX-659 and VX-445) in phase III studies as part of a triple combination with tezacaftor and ivacaftor. It plans to file regulatory submissions for one of the two regimens and has chosen VX-445 triple combination regimen for regulatory submissions in 2019. If the triple-combo regimen is approved, Vertex can address a significantly larger CF patient population – almost 90% of patients with CF - in the future.

Vertex currently has a Zacks Rank #2 (Buy). Some other top-ranked stocks from the biotech sector include Anika Therapeutics Inc. ANIK, Merus N.V. MRUS and Acorda Therapeutics, Inc. ACOR, all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Anika’s earnings estimates have been revised 8.3% upward for 2019 and 9.9% for 2020 over the past 60 days. The stock has gained 19.9% so far this year.

Merus’ shares have gained 1.8% this year so far. Its loss estimates have narrowed by 22.2% for 2019 and 17.2% for 2020 over the past 60 days.

Acorda’s loss estimates have narrowed 6.5% for 2019 and 6.9% for 2020 over the past 60 days.

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