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Vertex (VRTX) CF Drug Gets FDA Expanded Label for Toddlers

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Vertex Pharmaceuticals (VRTX - Free Report) announced that the FDA approved the expanded use of its cystic fibrosis (CF) drug Orkambi (lumacaftor/ivacaftor) in children aged 12 months to less than 24 months.

The approval covers treatments of CF in toddlers who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The approval for Orkambi is based on data from a phase III study that evaluated the drug in participants aged one year to less than two years over a 24-week period. Data from the study showed that participants treated with Orkambi demonstrated a safety profile and pharmacokinetics identical to those observed in patients aged two years and older who were treated with the drug.

Additional data from the study also showed that Orkambi has the potential for CF disease modification, including a reduction in sweat chloride concentration.

Prior to label expansion, Orkambi was already approved by the FDA to treat CF in people aged two years and older with two copies of the F508del mutation.

Vertex expects that the label expansion will allow the company to treat nearly 300 children suffering from CF.

Shares of Vertex have risen 28.0% in the year-to-date period against the industry’s 24.3% decline.

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CF is a rare genetic disorder that affects multiple organs, including the heart, liver and lungs. It is caused by the absence or shortage of CFTR protein, resulting in poor flow of salt and water into and out of the cells in several organs.

Such consistent positive regulatory approvals have led to an increase in the eligible patient population for Vertex’s approved medicines in the past 2-3 years. All these approvals have led to an increase in product revenues for Vertex.

Currently, CF is the key focus of Vertex. The company’s lead marketed products are Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko/Symkevi (tezacaftor/ivacaftor and ivacaftor), Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor), which are collectively approved to treat more than 83,000 people with CF in North America, Europe and Australia. In first-half 2022, the company generated $4.3 billion entirely as revenue from CF product sales, up 22% from the year-ago period’s levels.

 

Zacks Rank & Stocks to Consider

Vertex currently carries a Zacks Rank #3 (Hold). Some better-ranked stock in the overall healthcare sector includes Agenus (AGEN - Free Report) , Morphic (MORF - Free Report) and Sesen Bio (SESN - Free Report) , each of which has a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 30 days, estimates for Agenus’ 2022 loss per share have narrowed from 89 cents to 70 cents. Loss estimates for 2023 have narrowed from 64 cents to 60 cents during the same period. Shares of Agenus have lost 20.2% in the year-to-date period.

Earnings of Agenus beat estimates in three of the last four quarters and missed the mark just once, witnessing a surprise of 12.02%, on average. In the last reported quarter, AGEN delivered an earnings surprise of 26.09%.

In the past 30 days, estimates for Morphic’s 2022 loss per share have narrowed from $2.38 to $1.75. Loss estimates for 2023 have narrowed from $3.87 to $3.77 during the same period. Shares of Morphic have lost 40.2% in the year-to-date period.

Earnings of Morphic beat estimates in three of the last four quarters and missed the mark just once, witnessing a surprise of 48.29%, on average. In the last reported quarter, MORF delivered an earnings surprise of 183.95%.

Estimates for Sesen Bio’s 2023 bottom line have narrowed from 27 cents to 1 cent in the past 30 days. Share prices of Sesen Bio have fallen 19.0% in the year-to-date period.

Earnings of Sesen Bio beat estimates in each of the last four quarters, the average surprise being 89.49%. In the last reported quarter, Sesen Bio delivered an earnings surprise of 61.54%.

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