LONDON, UK — Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “Thousands of people with CF...
treatment News
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data from multiple studies demonstrating the clinical benefits of CASGEVY® (exagamglogene autotemcel) in people ages 5 years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, including the first presentation of clinical data from pivotal...
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...
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...
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)...