UK Approves First Crispr Gene Edited Therapy For Sickle Cell Disease, Beta Thalassemia

The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

Vertex Pharmaceuticals Incorporated VRTX jointly developed the gene therapy with CRISPR Therapeutics AG CRSP.

There are an estimated 2,000 patients eligible for Casgevy in the U.K.

"Today is a historic day in science and medicine: this authorization of Casgevy in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.

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. 

The safety profile of 97 SCD and TDT patients treated to date with Casgevy in these ongoing studies is generally consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.

Financial Times notes that the drug could replace bone marrow transplants.

The report adds that Casgevy is a difficult drug to administer. Patients must have their stem cells extracted from their bone marrow so their genes can be edited in a laboratory. After the edited cells are put back into their body, patients need to spend at least a month in hospital before they begin to make normal red blood cells. 

Price Action: VRTX shares closed at $349.34 on Wednesday, and CRSP shares are up 4.93% at $59.00 during the premarket session on the last check Thursday.