On December 8, the Food and Drug Administration “approved the country’s first gene-editing treatment, Casgevy, for use in patients with sickle cell disease. It will cost $2.2 million per patient per treatment, reports NBC. Later that same day, the FDA approved another sickle-cell drug called Lyfgenia. It will cost $3.1 million per patient per treatment. If all of the tens of thousands of people who could benefit from the treatment get it, that will cost tens of billions of dollars, much of it presumably covered by taxpayers, directly or indirectly. Half of all U.S. healthcare spending is by federal, state, or local governments.
But for people in agonizing pain from sickle cell disease, the cost will presumably be worth it. NBC reports that
the approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement….Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses Nobel Prize-winning technology CRISPR to edit a person’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 become misshapen half moons that get stuck inside blood vessels, restricting blood flow and causing what are known as pain crises. About 100,000 Americans are estimated to have the disease.
Casgevy uses CRISPR to make an edit to a person’s DNA that turns on fetal hemoglobin, a protein that normally shuts off shortly after birth, to help 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 are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease,” said Dr. Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, in a statement….
While the treatment itself is administered only once, the whole 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 a few days to clear out the old cells and make room for the new ones. After the 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 people with severe cases of sickle cell will be eligible….Because the procedure is so complex, it will be limited to certain health facilities like academic medical centers. Nine health-care facilities are ready to start administering Casgevy, Vertex said in a release, with more facilities added in the coming weeks….
The FDA also on Friday approved a separate gene therapy by Bluebird Bio, called Lyfgenia that works differently than Casgevy but is administered similarly and is also intended to eliminate pain crises. That therapy was similarly approved for the treatment of sickle cell disease in people 12 years and older.
Bluebird will charge $3.1 million per patient for Lyfgenia. Shares of that company, which has a market value of just about $300 million, fell 40% Friday.
In other news, British doctors say a new course of treatment for cervical cancer could cut cervical cancer deaths by 35%. Artificial intelligence is now developing highly-effective antibodies to fight disease.
Scientists recently came up with an “inverse vaccine” that has shown it can treat auto-immune diseases in a lab setting, so doctors might be able to use it to reverse multiple sclerosis. Note, however, that the FDA can take many years to approve life-saving drugs and medical devices.
Doctors recently used a surgical robot to carry out incredibly complicated spinal surgery, and used a robot to do a liver transplant. Robots can fit in small spaces in people’s bodies that a surgeon can’t reach without cutting through living tissue.
90% of those with cystic fibrosis will get a new lease on life with a new breakthrough drug, assuming the FDA doesn’t block it.
A new ultrasound therapy could help treat cancer and Alzheimer’s disease and skull implants could fight depression.
Artificial wombs could be coming soon, to prevent premature babies from dying or being permanently disabled due to premature life outside the womb.
