Casgevy is a genome-edited cellular therapy that consists of autologous CD34+ HSCs edited by CRISPR/Cas9 technology, while Lyfgenia is a cell-based gene therapy that uses lentiviral vector for genetic modification
FDA has approved Casgevy and Lyfgenia for SCD. (Credit: Sangharsh Lohakare on Unsplash)
Vertex Pharmaceuticals and CRISPR Therapeutics have received US Food and Drug Administration (FDA) approval for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease (SCD).
Casgevy is a one-time genome-edited cellular therapy that consists of autologous CD34+ hematopoietic stem cells (HSCs) edited by CRISPR/Cas9 technology.
The FDA has approved Casgevy for 12 years and older SCD patients with recurrent vaso-occlusive crises (VOCs).
The approval was based on data from an ongoing single-arm, multi-centre trial in adult and adolescent patients with SCD.
Out of 31 patients, 29 of them were free from severe VOC episodes for at least 12 consecutive months during the 24-month follow-up period.
In addition, all treated patients showed successful engraftment with no patients experiencing graft rejection or graft failure.
Vertex CEO and president Reshma Kewalramani said: “Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US.
“As importantly, Casgevy is a first-in-class treatment that offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease.”
The US health regulator has also granted approval to Bluebird Bio for Lyfgenia for SCD treatment in patients aged 12 years and more.
Lyfgenia is a cell-based gene therapy that uses lentiviral vector for genetic modification.
Its approval was supported by results from a single-arm, 24-month multicentre study. The effectiveness was evaluated based on the complete resolution of VOEs between six and 18 months post-infusion.
According to the findings, 28 of 32 patients completed VOE-CR during this period.
FDA Center for Biologics Evaluation and Research director Peter Marks said: “These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health.
“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating the development of safe and effective treatments for conditions with severe impacts on human health.”