Jan 16, 2024 · BOSTON -- (BUSINESS WIRE)--Jan. 16, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in …

Dec 8, 2023 · Vertex has submitted a BLA to the U.S. FDA for the potential use of CASGEVY for patients 12 years and older with TDT and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 30, 2024.

Dec 8, 2024 · BOSTON--(BUSINESS WIRE)--Dec. 8, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data for CASGEVY™ (exagamglogene autotemcel) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). CASGEVY is the first and only approved CRISPR/Cas9 gene ...

CASGEVY is indicated for the treatment of patients aged 12 years and older with: Monitor absolute neutrophil counts (ANC) and manage infections according to standard guidelines and medical judgement. In the event of neutrophil engraftment failure, patients should be infused with rescue CD34+ cells. Delayed Platelet Engraftment.

CASGEVY is an autologous genome edited hematopoietic stem cell-based gene therapy indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso ...

Jun 14, 2024 · Six months after Vertex earned a historic FDA approval for sickle cell disease (SCD) gene therapy Casgevy (exa-cel), long-term data is beginning to show the consistent efficacy and durability...

Nov 16, 2023 · In two global clinical trials of CASGEVY in SCD and TDT, the trials met their respective primary outcome of becoming free from severe VOCs or transfusion independent for at least 12 consecutive months. Once achieved, these benefits are potentially expected to …

Jan 16, 2024 · Vertex Pharmaceuticals and CRISPR Therapeutics have scored an FDA approval for their gene-editing therapy Casgevy (exa-cel) to treat transfusion-dependent beta thalassemia (TDT). The approval...

1 day ago · LONDON, January 31, 2025--Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 ...

Nov 5, 2024 · With the first Casgevy revenues coming in and the company's established cystic fibrosis portfolio firing on all cylinders, Vertex brought in a quarterly haul of $2.77 billion, a 12% increase...