-Exa-cel is the first CRISPR-based gene-edited therapy to be submitted for Health Canada review-

TORONTO, April 1, 2024 /CNW/ - 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 treatment of patients aged 12 years and older with transfusion-dependent beta thalassemia (TDT).

"We are pleased that exa-cel has been accepted for Priority Review by Health Canada and look forward to bringing this therapy to eligible patients," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated.

The NDS will be part of an aligned review with Health Technology Assessment (HTA) organizations, the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Quebec.

With Priority Review, the conventional review timeline of 300 days is reduced to 180 days.

The NDS is supported by results from the ongoing Phase 3 studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study, CLIMB-131. Data from the Phase 3 studies were most recently presented at the American Society of Hematology (ASH) Annual Meeting and Exposition in December of 2023.