Health Canada has approved Casgevy, a gene-edited cell therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics, for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in patients aged 12 years and older.

Casgevy, also known as exagamglogene autotemcel (exa-cel), received FDA approval in December 2023 for the same indications, marking it as the first CRISPR-based gene-editing therapy approved in Canada.

This approval is a significant advancement for patients suffering from these conditions, as noted by Michael Siauw, general manager at Vertex Pharmaceuticals (Canada) Incorporated.

Casgevy works by increasing fetal hemoglobin production to prevent the sickling of red blood cells, addressing the underlying cause of sickle cell disease.

The therapy utilizes patients' own blood stem cells and requires a one-time infusion following myeloablative conditioning.

Clinical trial data from the ongoing CLIMB-121 and CLIMB-111 studies, conducted in multiple countries, supported the approvals, with both trials meeting their primary endpoints.

In the CLIMB-111 trial, 93% of evaluable patients with TDT were free from the need for red blood cell transfusions for 12 months after treatment.

Dr. Melissa Creary, who has sickle cell disease herself, expresses excitement about the new treatments but also highlights potential barriers such as cost, accessibility, and treatment complexity.

Experts emphasize the critical role of healthcare providers in guiding patients through treatment options, considering both short-term and long-term risks and benefits.

Despite its approval, the rollout of Casgevy has been slow, with no patients treated within the first six months post-launch, indicating challenges in implementation.

Vertex is collaborating with Charles River Laboratories for the production of Casgevy at a facility in Memphis, Tennessee, and has signed a long-term supply agreement with Lonza for additional manufacturing.

Ciaran Brady, VP of Manufacturing Science and Technology at Vertex, discussed the need for a flexible manufacturing network to avoid overcapacity and maintain profitability while expanding their global network of manufacturing sites.