FDA approves first gene therapies for sickle cell disease
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The FDA approved the first two cell-based gene therapies for treatment of sickle cell disease.
Both therapies — exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals), a CRISPR/Cas9 gene-edited therapy, and lovo-cel (Lyfgenia, Bluebird Bio) — are intended for treatment of patients aged 12 years or older.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” Nicole Verdun, MD, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, said in a press release. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”
Both therapies are made from patients’ own blood stem cells, which are modified and then returned via a one-time single infusion as part of a hematopoietic stem cell transplant. Patients who have received both therapies will be followed in a long-term study designed to further assess the effectiveness and safety of each product, according to the FDA.
Exagamglogene autotemcel, often called exi-cel, is indicated for treatment of patients with recurrent vaso-occlusive crises. It is the first FDA-approved therapy using CRISPR/Cas9, a form of genome editing technology.
“FDA approval of Casgevy for severe sickle cell disease marks a seminal moment in the history of biotechnology and human health,” Tim Hunt, CEO of Alliance for Regenerative Medicine, told Healio. “As the first gene-editing medicine approved in the United States, Casgevy represents a durable and potentially curative treatment option for a population that has been overlooked for far too long — and one that is the largest to date for a gene therapy. Importantly, [this] milestone will also pave the way for a coming wave of next-generation gene-editing treatments for a range of diseases, from other rare disorders to cancers.”
Lovo-cel — a cell-based gene therapy that uses a lentiviral vector for genetic modification — is indicated for patients with sickle cell disease and a history of vaso-occlusive events.
“The approval of the first gene therapies for sickle cell disease represents a tremendous step forward,” ASH President Robert A. Brodsky, MD, said in a society-issued press release.
The community of individuals with sickle cell disease “has been historically overlooked and underfunded,” Brodsky said.
“While these new gene therapies are potentially life-changing for individuals living with sickle cell disease, they must be accessible to be effective,” Brodsky said. “Despite the recent progress in expanding sickle cell disease therapies, current treatments and treatment models do not adequately address the many barriers to care people living with sickle cell disease face, including access to providers that understand their condition, hydroxyurea for symptom management, pain medication during pain crises, and specialized and high-quality care.”
References:
- ASH statement on FDA’s approval of new sickle cell disease gene therapy (press release). Available at: https://www.hematology.org/newsroom/press-releases/2023/ash-statement-on-fda-approval-of-new-sickle-cell-disease-gene-therapy. Published Dec. 8, 2023. Accessed Dec. 8, 2023.
- FDA approves first gene therapies to treat patients with sickle cell disease (press release). Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease. Published Dec. 8, 2023. Accessed Dec. 8, 2023.
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