Cystic fibrosis is the most common lung affecting genetic disease, which often turns out to be life-threatening. Nearly one in every 2500 to 3500 people are diagnosed with cystic fibrosis at an early age. A thick mucus formation in the lungs becomes stuck to bacteria, which leads to a slimy biofilm formation.
An IPO for raising USD 115 million from public investors has been filed by Translate Bio. Proceeds garnered from the IPO will be used by Translate to trial an mRNA candidate designed to allow cystic fibrosis patients produce CTFR proteins.
Vertex Pharmaceuticals Incorporated (VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor. SYMDEKO is Vertex’s third medicine approved to treat the underlying cause of CF. Vertex is ready to launch SYMDEKO and will begin shipping it to pharmacies in the United States this week.
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