Washington, December 8 (EFE).- The US Food and Drug Administration (FDA) announced this Friday that it has approved two treatments for sickle cell anemia (SCD), one of which is the first commercially available in the country based on genetics. Editing technology.
In a statement, the FDA announced the approval of Kaskavi and Lifgenia to treat the effects of this genetic disorder in which red blood cells become stiff and sticky when they change from a disc to a crescent shape.
Kaskawi is the first FDA-approved treatment using a new type of gene editing technology, which the company notes „represents an innovative advance in the field of gene therapy.”
Sickle cell anemia is a group of inherited blood disorders that affect about 100,000 people in the United States. Although it is more common among African Americans and less frequently, it also affects Hispanic Americans.
The main problem with sickle cell anemia is a change in hemoglobin, the protein found in red blood cells that carries oxygen to the body’s tissues.
Sickled red blood cells restrict flow in blood vessels and restrict oxygen delivery to the body’s tissues, causing severe pain and organ damage that can lead to life-threatening disabilities or premature death.
Two approved treatments are made from the patient’s own blood stem cells, which are modified and transplanted back.
Cascav is the first FDA-approved treatment to use CRISPR/Cas9, a type of gene editing technology that cuts DNA at specific regions, allowing DNA to be precisely edited (deleted, added, or replaced).
The safety and efficacy of Caskavi was evaluated in a trial of 44 patients with a history of serious adverse events and the primary efficacy outcome was absence of serious events for at least 12 months during a 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 provide highly specific and effective treatments for patients with specific rare diseases, particularly those for whom current treatment options are limited.”