Vertex expands the indication of its drug to combat cystic fibrosis. The Committee for Human Use (Chmp) of the European Medicines Agency (EMA) has issued a positive opinion for the extension of the indication for Kaftrio in combination with ivacaftor, expanding the range of treatment for cystic fibrosis in children from two to five years.
In the European Union, Kaftrio in combination with ivacaftor is already approved for the treatment of people with cystic fibrosis for children aged six years and older. If this positive opinion is ratified and use in children under five years of age is authorized, Vertex assures that more than 1,200 children would be candidates to receive a medication that could treat the underlying cause of their illness.
Sara Alonso, medical director of Vertex, noted that “treating the underlying cause of cystic fibrosis as soon as possible has the potential to slow the progression of the disease, which is why we welcome the fact that the Chmp supports the extension of the indication for Kaftrio to patients as young as two years,” in the words of the company director.
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In the European Union, Vertex’s drug is already approved for children six years and older
Cystic fibrosis is a genetic disease that affects more than 88,000 people worldwide. Specifically, in a progressive condition that damages multiple organs, including the lungs, liver, pancreas, or gastrointestinal tract. The disease is caused by a defective or missing Cftr (cystic fibrosis transmembrane conductance regulator) protein and life expectancy is approximately thirty years.
Vertex is a global biotechnology company that invests in scientific innovation to create medicines for people with serious diseases. The company has multiple medications approved to treat cystic fibrosis and several clinical and research programs underway to try to better understand the disease.
The company was founded in 1989 in Cambridge (Massachusetts). The global headquarters is located in Boston’s Innovation District and its international headquarters is located in London. In the financial results for the second quarter of the year, the firm reported product revenues of $2.49 billion, an increase of 14% compared to the same period of the previous year. In its forecasts, Vertex predicts closing the year with revenues of $9.8 billion.
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First published in PlantaDoce. A third-party contributor translated and adapted the article from the original. In case of discrepancy, the original will prevail.
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