BOSTON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that in a Phase 2 proof-of-concept study, VX-864 achieved rapid, consistent and statistically significant increases in mean functional alpha-1 antitrypsin (fAAT) levels of 2.2 to 2.3 micromolar from baseline in people with alpha-1 antitrypsin deficiency (AATD) with the PiZZ genotype, across...
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BOSTON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene...
BOSTON, Mass. – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the presentation of multiple abstracts demonstrating the clinical benefits of treatment with CFTR modulators, including the Company’s most recently approved medicine, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor), at the North American Cystic Fibrosis Conference (NACFC) held from October 22-25 in Seattle, Washington. Several of the abstracts that will be presented at...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced updated data for povetacicept (pove) in IgA nephropathy (IgAN) and primary membranous nephropathy (pMN) from the ongoing RUBY-3 trial at the American Society of Nephrology (ASN) Kidney Week 2025 in Houston, Texas. Pove is an investigational recombinant fusion protein therapeutic and dual inhibitor of...
LONDON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF) ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)...
ALYFTREK®(deutivacaftor/tezacaftor/vanzacaftor) recommended for people with CF ages 6 years and older with at least one non-class I mutation in the CFTR gene – – In head-to-head clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior on ppFEV1 and superior compared to KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) at reducing sweat chloride – LONDON, UK –Vertex Pharmaceuticals (Nasdaq: VRTX) today announced...
LONDON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination with ivacaftor 150 mg tablets for the treatment of cystic fibrosis (CF) in all patients ages 12...
TORONTO, Canada – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced its New Drug Submission (NDS) for exagamglogene autotemcel (exa-cel) has been accepted for Priority Review by Health Canada for the treatment of patients aged 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for the...
AURORA, Colo.– Researchers at the University of Colorado Anschutz Medical Campus and collaborators across the country have conducted the largest and most diverse study of men with extra X or Y chromosomes in the US using a large dataset of military veterans. The results could lead to better diagnoses of...
SOUTH SAN FRANCISCO, Calif. — Vevo Therapeutics, a biotechnology company using its Mosaic technology and next generation AI to uncover better drugs for more patients, announced today that it has generated the world’s largest single-cell atlas of how drugs interact with and affect tumor cells. The company’s Mosaic platform is...