BREAKING | Vertex and CRISPR’s Gene-editing Therapy Casgevy Receives Early FDA Approval for β-Thalassaemia Treatment

 On Tuesday, January 16, 2024, the FDA gave the green light to Vertex Pharmaceuticals and CRISPR Therapeutics’ groundbreaking Casgevy™ (exagamglogene autotemcel) Cas9 gene-edited cell therapy. This one-time treatment is now approved for transfusion-dependent β-thalassaemia patients aged 12 and up.

Just one month prior, Casgevy made history as the first CRISPR gene editing therapy to enter the US market when it was approved for sickle cell disease. Now, the FDA has expanded its use for transfusion-dependent β- thalassaemia (TDT), more than two months ahead of schedule!

Originally, Vertex and CRISPR Therapeutics anticipated a decision by March 30, 2024. “Following the groundbreaking approval of Casgevy for sickle cell disease, it’s thrilling to see it approved for TDT well before the PDUFA date,” said Vertex CEO Reshma Kewalramani. “TDT patients need new, potentially curative treatments, and we can’t wait to bring Casgevy to those eagerly awaiting help.”

Vertex is collaborating with hospitals to create a network of authorized treatment centers (ATCs) in the US that will offer this life-changing therapy. More ATCs will be available in the coming weeks, and currently, nine centers can provide Casgevy for TDT and sickle cell disease patients across the country.

Utilizing the groundbreaking CRISPR/Cas9 gene editing system — a Nobel prize-winning technology—Casgevy modifies patients’ hematopoietic (blood) stem cells. The approval showcases the power and versatility of the CRISPR platform, offering a potentially curative option for those suffering from this devastating illness.

Aside from FDA approval, CASGEVY™ has gained approval for transfusion-dependent thalassaemia by the UK’s Medicines & Healthcare products Regulatory Agency – MHRA (Nov. 2023), the NHRA (Bahrain, Dec. 2023), the SFDA (Saudi Arabia, Jan. 2024) and a positive opinion from the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) (Dec. 2023). The final decision from the European Commission is expected in February 2024.

In terms of cost, Vertex has yet to reveal Casgevy’s list price for TDT treatment, but it’s worth noting that bluebird bio’s FDA-approved β-thalassaemia treatment, Zynteglo, costs $2.8 million.

Sources: Vertex Pharmaceuticals Press Release, CRISPR Therapeutics Press Release

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