collected by :Molly Tony
Vertex Pharmaceuticals Inc. is closer to being able to sell its third drug to treat cystic fibrosis.
Each of them have one of about 20 rare genetic defects along with the so-called F508del mutation that is common in other CF patients.
Sign up for more newsletters here“We’ve cracked the code of cystic fibrosis,” Vertex chief executive Jeffrey Leiden said, citing data that demonstrated the combination therapy was safe and effective.
Both studies show statistically significant improvement in two different sets of CF patients, including one group — those with copies of two different disease-causing genetic mutations — for which there aren’t any medicines.
To treat the rest, the company is developing new combination drugs that can work effectively in sets of patients with different genetic defects.
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Vertex rockets on cystic fibrosis results
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as declared in Kalydeco, known chemically as ivacaftor, was given every 12 hours, while tezacaftor, previously known as VX-661, was taken once a day.
Vertex Pharmaceuticals said on Tuesday its Kalydeco cystic fibrosis treatment givenwith an experimental drug demonstrated significant improvements in lung function in a pair of late-stage trials the company plans to use to seek approval for the combination therapy.
The studies tested CF patients aged 12 and over.
Vertex’s approved medicines address about 29,000 CF patients.
It is testing drugs for a triple combination with Kalydeco and tezacaftor that could eventually treat 80-90 percent of all CF patients, with data expected later this year.