FDA Approves Vertex’s CF Drug for Toddlers

Vertex Pharmaceuticals NASDAQ:VRTX

Vertex Pharmaceuticals (NASDAQ:VRTX)

Orkambi (lumacaftor/ivacaftor), manufactured by Vertex Pharmaceuticals (NASDAQ:VRTX), has received FDA approval for the treatment of cystic fibrosis (CF) in children aged 12 months to less than 24 months.

Treatments for infants and toddlers with CF who carry two copies of the F508del mutation in the CFTR gene have been given the green light.

Orkambi was approved based on the results of a phase III research that looked at the drug’s effects over 24 weeks in people aged one to under two. Researchers found that the safety profile and pharmacokinetics of Orkambi in this study’s participants were consistent with those seen in patients aged 2 and up.

The study’s further findings, including a decrease in sweat chloride content, support the idea that Orkambi may help modify CF illness.

Orkambi was previously FDA-approved to treat CF in patients two years and older with two copies of the F508del mutation before the label was expanded.

As a result of the label extension, Vertex hopes to treat 300 more CF-afflicted youngsters.

Vertex Pharmaceuticals (NASDAQ:VRTX) year-to-date share price increase of 28.0% compares well to the industry’s decrease of 24.3%.

The heart, liver, and lungs are just some of the organs that can be negatively impacted by CF, making it an uncommon hereditary illness. Due to a lack of CFTR protein, salt and water have trouble moving into and out of cells in several organs.

Over the past two to three years, the number of people who can take Vertex’s licensed medications has grown thanks to a string of positive regulatory approvals. Vertex’s product sales have improved due to all these green lights.

Vertex Pharmaceuticals (NASDAQ:VRTX) is now concentrating its efforts on CF. Across North America, Europe, and Australia, the company’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko/Symkevi (tezacaftor/ivacaftor and ivacaftor), Orkambi (lumacaftor/ivacaftor), and Kalydeco (ivacaftor) are approved to Sales of CF products accounted for 100% of the company’s $4.3 billion in revenue in the first half of 2022, an increase of 22% over the same time the previous year.

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