Previously known as CTX-001, Casgevy is a non-viral ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease or TDT
US FDA approves Casgevy for beta-thalassemia. (Credit: DIGITALE on Unsplash)
Vertex Pharmaceuticals has received the US Food and Drug Administration (FDA) approval for Casgevy (exagamglogene autotemcel [exa-cel]) to treat transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.
Previously known as CTX-001, Casgevy is a non-viral ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or TDT.
The drug edits a patient’s own hematopoietic stem and progenitor cells at the erythroid specific enhancer region of the BCL11A gene via a precise double-strand break.
Vertex president and CEO Reshma Kewalramani said: “On the heels of the historic FDA approval of Casgevy for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date.
“TDT patients deserve new, potentially curative treatment options, and we look forward to bringing Casgevy to eligible patients who are waiting.”
In November last year, Vertex and CRISPR Therapeutics secured conditional marketing authorisation for Casgevy in the UK to treat SCD and TDT.
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for eligible patients 12 years of age and older.
The therapy is intended for patients with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not accessible.
Vertex said the approval was based on two global clinical trials of Casgevy in SCD and TDT.
Both the studies met their respective primary outcome of becoming transfusion independent or free from severe volatile organic compounds (VOCs) for a minimum of 12 consecutive months.
According to Vertex, 97 SCD and TDT patients who have received treatment with Casgevy thus far in the ongoing trials have a safety profile that was consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.
The company has secured an Innovation Passport for the drug, under MHRA’s Innovative Licensing and Access Pathway (ILAP) and is working closely with national health authorities.