Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that the US Food and Drug Administration (FDA) has approved CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

This approval means that for the first time, approximately 16,000 patients with SCD may be eligible for a durable one-time therapy that offers the potential of a functional cure for their disease by eliminating severe VOCs and hospitalizations caused by severe VOCs.

“CASGEVY’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the U.S. 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,” said Reshma Kewalramani, Chief Executive Officer and President of Vertex.

“I want to convey my deepest gratitude to the patients and investigators whose trust in this program paved the way for this landmark approval.”

“When our company was founded, we had a vision to translate CRISPR technology into multiple breakthrough therapies. So, this U.S. approval of the first-ever medicine using CRISPR gene editing is breathtaking, and a truly humbling moment for me personally and for the whole organization,” said Samarth Kulkarni, Chairman and Chief Executive Officer of CRISPR Therapeutics. 

“It has been remarkable to be part of this groundbreaking program,” said Stephan Grupp, M.D., Section Chief of the Cellular Therapy and Transplant Section and Director of the Kelly Center for Cancer Immunotherapy at the Children's Hospital of Philadelphia, and Steering Committee Chair for the CLIMB-121 clinical program. “CASGEVY has the potential to be a transformative treatment for patients, and I look forward to continuing the work to ensure eligible patients can access this therapy across the country.”

The administration of CASGEVY requires specialized experience in stem cell transplantation; therefore, Vertex is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to patients. The following ATCs are already activated: