LONDON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced it has reached a new portfolio agreement with the Italian Medicines Agency, AIFA, for the reimbursement of all of Vertex’s approved medicines for the treatment of cystic fibrosis (CF), including KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor. Italian patients ages 12 years...
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BOSTON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that, in a Phase 2 proof-of-concept (POC) study in patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS), VX-147 on top of standard of care achieved a statistically significant, substantial and clinically meaningful mean reduction of 47.6% in the urine protein to creatinine...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its once-daily vanzacaftor/tezacaftor/deutivacaftor (the “vanza triple”) program, the most comprehensive Phase 3 pivotal program ever conducted by Vertex for the treatment of cystic fibrosis (CF), a progressive, multi-organ disease caused by dysfunction of the CFTR protein. The...
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...
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...
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)...
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...
LEIDEN, Netherlands — Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced a $60 million (€54 million) Series B financing. The financing was led by new investor Ackermans & van Haaren (AvH) with existing co-leads Droia Ventures, EQT Life Sciences and Kurma Partners...