![FDA greenlights first gene-editing treatment, Casgevy, for sickle cell disease](https://image.cnbcfm.com/api/v1/image/107345254-17020557071702055705-32366157981-1080pnbcnews.jpg?v=1702055706&w=750&h=422&vtcrop=y)
The U.S. Food and Drug Administration on Friday approved the country’s first gene-editing treatment, Casgevy, to be used in patients with sickle cell disease.
The approval comes a few decade after the invention of CRISPR technology for editing human DNA, representing a big scientific advancement. Yet reaching the tens of hundreds of people that may benefit from the treatment may very well be difficult given the potential hurdles — including cost — of administering the complex therapy.
Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses Nobel Prize-winning technology CRISPR to edit an individual’s genes to treat disease. The treatment was approved by U.K. regulators last month.
Sickle cell, an inherited blood disorder, causes red blood cells to turn out to be misshapen half moons that get stuck inside blood vessels, restricting blood flow and causing what are referred to as pain crises. About 100,000 Americans are estimated to have the disease.
This microscope photo provided on Oct. 25, 2023, by the Centers for Disease Control and Prevention shows crescent-shaped red blood cells from a sickle cell disease patient in 1972. Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that would offer relief to hundreds of individuals with the crippling disease in the U.K.
Dr. F. Gilbert/CDC via AP, File
Casgevy uses CRISPR to make an edit to an individual’s DNA that activates fetal hemoglobin, a protein that normally shuts off shortly after birth, to assist red blood cells keep their healthy full-moon shape. In clinical trials, Casgevy eliminated pain crises in most patients.
The FDA approved the treatment for people 12 years and older.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we’re excited to advance the sector especially for people whose lives have been severely disrupted by the disease,” said Dr. Nicole Verdun, director of the Office of Therapeutic Products inside the FDA’s Center for Biologics Evaluation and Research, in a press release.
“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for people with rare diseases where the present treatment options are limited,” Verdun added.
While the treatment itself is run just once, the entire process takes months. Blood stem cells are extracted and isolated before being sent to Vertex’s lab, where they’re genetically modified. Once ready, patients receive chemotherapy for just a few days to filter the old cells and make room for the brand new ones. After the brand new cells are infused, recipients spend weeks in the hospital recovering.
Vertex will take the lead on launching the drug and estimates about 16,000 individuals with severe cases of sickle cell shall be eligible.
Even among the many individuals who may benefit probably the most, analysts worry few will clamor for a treatment that takes months to finish, carries the chance of infertility and may very well be cost prohibitive.
Vertex will charge $2.2 million per patient, the corporate said in a regulatory filing.
And, since the procedure is so complex, it can be limited to certain health facilities like academic medical centers. Nine health-care facilities are ready to begin administering Casgevy, Vertex said in a release, with more facilities added in the approaching weeks.
The FDA also on Friday approved a separate gene therapy by Bluebird Bio, called Lyfgenia that works in another way than Casgevy but is run similarly and can also be intended to eliminate pain crises. That therapy was similarly approved for the treatment of sickle cell disease in people 12 years and older.
It is a developing story. Please check back for updates.