The revolutionary CRISPR gene editing is now fully part of the arsenal of medicine. The United States Food and Drug Administration (FDA) today announced the approval of Casgevy, the first treatment that uses a type of novel genome editing technology, CRISPR, according to the agency. It is used for the treatment of sickle cell anemia in patients over 12 years of age. Along with Casgevy, the regulator has approved the use of another cellular gene therapy, Lyfgenia, in this case conventional.
The treatment was submitted in October to the demanding examination of the US regulator at the request of the Boston-based laboratories Vertex Pharmaceuticals and the Swiss CRISPR Therapeutics. Together they have designed a therapy based on genome editing against a painful blood disease: sickle cell anemia, a genetic malformation responsible for the deformation of red blood cells. It is one of the most frequent in the world, and affects millions of people, 100,000 in the United States alone. It also promises to improve the outlook for patients with another congenital anemia: beta-thalassemia, which requires lifelong transfusions.
Sickle cell anemia is a group of inherited blood disorders. Its main problem is a mutation in hemoglobin, a protein found in red blood cells that supplies oxygen to the body’s tissues. This mutation causes the red blood cells, instead of the usual round shape, to acquire the silhouette of a crescent or sickle, which causes them to get stuck in the blood vessels and cause pain incompatible with the development of a normal life. Most patients do not live beyond 40 or 50 years.
For those affected, the approval of this treatment is a ray of hope. However, it is still an extraordinarily expensive and complex therapy to administer, and it will only be within the reach of a privileged few.
The safety and effectiveness of Casgevy were evaluated in a trial in adult and adolescent patients with SCD. Patients had a history of at least two serious episodes of vaso-occlusive incidents. The primary efficacy outcome was the absence of such serious events for at least 12 consecutive months during the 24-month follow-up period. A total of 44 patients were treated with Casgevy. Of the 31 patients with sufficient follow-up time to be evaluable, 29 (93.5%) achieved this result. All treated patients achieved a satisfactory graft and none experienced graft failure or rejection.
The most common side effects were low levels of platelets and white blood cells, mouth ulcers, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile neutropenia (fever and low white blood cell count), headache, and pruritus, according to the FDA.
“Sickle cell anemia is a rare, debilitating and life-threatening blood disorder with significant unmet needs, and we are pleased to advance this field, especially for people whose lives have been severely disrupted by the disease, by approving two gene therapies today. “cells,” said Dr. Nicole Verdun, director of the Office of Therapeutic Products at the FDA’s Center for Biologics Evaluation and Research, in a statement. “Gene therapy holds the promise of offering more specific and effective treatments, especially for people with rare diseases where current therapeutic options are limited.”
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