USA – The US Food and Drug Administration (FDA) has granted approval for the groundbreaking CRISPR-based gene editing therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
Casgevy (exagamglogene autotemcel) represents the first-ever CRISPR-based treatment for sickle cell disease (SCD).
This approval follows a similar milestone by its U.K. counterpart and marks a significant leap forward in the realm of gene therapies.
Casgevy, hailed as a potential cure for sickle cell disease affecting approximately 100,000 Americans, addresses a genetic mutation related to the production of hemoglobin beta, causing red blood cells to take on a characteristic “sickle” shape.
This abnormality leads to sticky blood cells, obstructing blood flow, and resulting in anemia and painful vaso-occlusive events.
The FDA approval specifically covers the treatment of SCD patients aged 12 and older experiencing recurrent vaso-occlusive crises.
The therapy employs the Nobel prize-winning CRISPR/Cas9 gene editing system, a revolutionary technology developed over a decade ago by Jennifer Doudna and Emmanuelle Charpentier.
Casgevy works by editing a patient’s hematopoietic stem cells to stimulate the production of high levels of fetal hemoglobin.
The approval is founded on data from a pivotal Phase I/II/III trial and a long-term safety and efficacy study, demonstrating significant reductions in severe vaso-occlusive events and hospitalizations.
Simultaneously, Bluebird bio’s Lyfgenia, a lentiviral gene therapy, also secured FDA approval. Lyfgenia introduces a functional copy of the hemoglobin beta gene directly into a patient’s hematopoietic stem cells.
A 24-month study revealed that 28 out of 32 patients treated with Lyfgenia achieved complete resolution of vaso-occlusive events.
Notable voices in the medical community, such as Martin Steinberg, a hematologist at Boston University and a member of Vertex’s exa-cel steering committee, have lauded the trial results as “spectacular” and even exceeding expectations.
Eric Kmiec, founder and executive director of the ChristianaCare Gene Editing Institute, emphasized the significance of a potential cure for sickle cell disease but highlighted the challenges of accessibility and affordability.
In an interview, Kmiec said, “The challenge, however, is the very people we want to help may not be able to get access to or afford the million-dollar treatment and the length of time it will take to be treated—weeks and weeks in the hospital.”
Tim Hunt, CEO of the Alliance for Regenerative Medicine (ARM), reflected on the historic approvals and the unmet need in sickle cell disease.
Hunt stated, “Today’s FDA approval of Casgevy for severe sickle cell disease marks a seminal moment in the history of biotechnology and human health.”
He emphasized that the approval opens the door for a new era of gene-editing treatments, not only for rare disorders but also for various diseases, including cancer.
The gene-editing landscape is rapidly advancing, with at least 120 clinical trials globally, 90 of which involve CRISPR technology.
Of these, 77% are targeting cancer indications. Vertex and CRISPR anticipate an FDA decision for Casgevy in transfusion-dependent beta-thalassemia by March 30, 2024.
The dual approvals signify a transformative moment in biotechnology, promising durable and potentially curative treatment options for a patient population that has long been underserved.
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