FDA Approves First Gene Editing Therapy Priced at $2.2 Million

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FDA Approves First Gene Editing Therapy Priced at $2.2 Million

FDA Approves First Gene Editing Therapy Priced at $2.2 Million

In a milestone event in the history of gene editing therapy, approximately a decade after the advent of CRISPR gene editing technology, a significant scientific advancement promises to benefit tens of thousands of patients.

On December 8, the U.S. Food and Drug Administration (FDA) granted approval for the country’s first gene editing therapy drug, Casgevy.

This marks a historic moment in the history of gene editing therapy, symbolizing a major scientific progress that could benefit numerous patients.

Casgevy, jointly developed by pharmaceutical company Vertex Pharmaceuticals and biotechnology company CRISPR Therapeutics, is designed for the treatment of sickle cell disease in adults aged 12 and above.

CRISPR Therapeutics, founded by Nobel Prize laureate Emmanuelle Charpentier, received approval from the UK regulatory authority for this therapy last month.

FDA Approves First Gene Editing Therapy Priced at $2.2 Million

One Treatment, Lifelong Cure

Sickle cell disease is a hereditary blood disorder where a gene mutation causes red blood cells, typically round, to form a crescent shape, getting stuck in blood vessels, restricting blood flow, and causing severe pain episodes.

“Sickle cell disease is a rare and life-threatening blood disorder with unmet needs, and we are pleased to advance developments in this field, especially for individuals whose lives are significantly disrupted by this disease,” stated the FDA’s Center for Biologics Evaluation and Research Office of Tissues and Advanced Therapies in a press release.

However, the cost of this medication is remarkably high, with a treatment cost of $2.2 million per patient. Based on Vertex’s current drug manufacturing capacity, approximately 16,000 severe sickle cell patients would qualify for the medication.

Despite the fact that this gene editing treatment only needs to be administered once, the entire preparation process takes several months. Specifically, the patient’s blood stem cells are first extracted and separated, then sent to Vertex laboratories for gene modification. After gene editing, patients undergo several days of chemotherapy to clear out old cells and make room for the new cells. After the infusion of the new cells, recipients need to spend several weeks in the hospital for recovery.

Reshma Kewalramani, CEO of Vertex, commented, “We believe that the price of the drug can reflect its value, and the value it brings is a one-time therapy that could potentially cure the disease for a lifetime.”

Meanwhile, on the same day, Bluebird Bio’s gene therapy Lyfgenia also received FDA approval for the treatment of sickle cell disease in individuals aged 12 and above, with the treatment cost soaring to $3.1 million per patient.

In 2012, scientists Jennifer Doudna and Emmanuelle Charpentier published a groundbreaking paper on the CRISPR-Cas9 gene editing system, leading to them being awarded the Nobel Prize. This discovery prompted numerous companies to explore the use of CRISPR gene editing technology for treating various diseases, with sickle cell disease being a primary target. Editing patient genes through CRISPR technology can activate the so-called “fetal hemoglobin” to help red blood cells maintain a healthy shape.