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Approximately 16,000 patients with sickle cell disease could be eligible for a one-time therapy that may offer a functional cure for their disease. This therapy has the potential to eliminate severe VOCs and hospitalizations caused by severe VOCs. Earlier this month, the UK’s MHRA authorized the the CRISPR/Cas9 treatment for sickle cell disease and transfusion-dependent beta thalassemia in the UK.
Vertex Pharmaceuticals Inc. and CRISPR Therapeutics have announced that the FDA has approved CASGEVY (exagamglogene autotemcel), a CRISPR/Cas9 genome-edited cell therapy for the treatment of sickle cell disease (SCD). Treatment is intended for patients 12 years and older with recurrent vaso-occlusive crises (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, M.D., CEO 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.”
Vertex is partnering with experienced hospitals to create a network of authorized treatment centers (ATCs) across the United States that will offer CASGEVY to patients. The administration of CASGEVY requires expertise in stem cell transplantation, so these ATCs will be independently operated and authorized to provide specialized treatment. Currently, the following ATCs are already activated:
- Boston Medical Center in Boston, Mass.
- Children’s National Hospital in Washington, D.C.
- City of Hope Children’s Cancer Center in Los Angeles, Calif.
- Medical City Children’s Hospital in Dallas, Texas
- Methodist Children’s Hospital in San Antonio, Texas
- Nationwide Children’s Hospital in Columbus, Ohio
- The Children’s Hospital at TriStar Centennial in Nashville, Tenn.
- The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute in Columbus, Ohio
- University of Chicago/Comer Children’s Hospital in Chicago, Ill.
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“It has been remarkable to be part of this groundbreaking program,” said Stephan Grupp, M.D., Ph.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.”
Additional ATCs will be activated in the coming weeks and a complete list of ATCs, including updates following approval, can be accessed at CASGEVY.com.
Source: Vertex Pharmaceuticals Inc.
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