The United States Food and Drug Administration (FDA) announced this Friday the approval of two treatments for sickle cell anemia (SCD), one of which is the first commercially available treatment in the country based on gene editing technology.
Through a statement, the FDA reported the approval of Casgevy and Lyfgenia to counteract the effects of this genetic disorder in which red blood cells become rigid and sticky when changing from a disk shape to a crescent shape.
Casgevy is also the first FDA-approved treatment that uses a novel type of genome editing technology, “indicating a groundbreaking advance in the field of gene therapy,” the agency notes.
Sickle cell anemia is a group of inherited blood disorders that affect approximately 100,000 people in the United States. It is more common among African Americans and, although less prevalent, it also affects Hispanic Americans.
The main problem with sickle cell anemia is a mutation in hemoglobin, a protein found in red blood cells that supplies oxygen to the body’s tissues.
Sickle red blood cells restrict flow in blood vessels and limit oxygen delivery to the body’s tissues, causing severe pain and organ damage, which can lead to life-threatening disabilities or premature death.
The two approved treatments are made from the patient’s own blood stem cells, which are modified and reimplanted.
For US President Joe Biden, today’s announcement “holds tremendous promise for the development of additional life-saving treatments and gives hope to the millions of Americans living with other rare diseases.”
As noted in a statement released by the White House, the FDA’s validation of these two methods “represents the power of medical innovation to improve the lives of Americans.”
Living with sickle cell anemia, he added, has often meant “putting the goals and plans of daily life on hold because of the chronic pain and serious health complications associated with it.”
Casgevy is the first FDA-validated therapy to use CRISPR/Cas9, a type of genome editing technology that can be used to cut DNA in specific areas, allowing DNA to be precisely edited (deleted, added, or replaced).
The safety and effectiveness of Casgevy was evaluated in a trial with 44 patients who had a serious history and the primary efficacy outcome was the absence of serious events for at least 12 consecutive months during the 24-month follow-up period.
According to Nicole Verdun, director of the Office of Therapeutic Products at the FDA’s Center for Biologics Evaluation and Research, gene therapy “promises to offer more specific and effective treatments, especially for people with rare diseases where current treatment options are limited.”
