FDA APPROVES FIRST GENE THERAPIES TO TREAT PATIENTS WITH SICKLE CELL DISEASE

The FDA has recently approved two gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease in patients 12 years and older. Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is based on the CRISPR gene editing technology, while Lyfgenia, developed by bluebird bio, uses a different approach by inserting modified genes into the body through disabled viruses. 

Sickle cell disease is an inherited blood disorder characterized by abnormal hemoglobin, which causes red blood cells to become rigid and sickle-shaped, leading to various complications such as pain, organ damage, and increased risk of infections.  

These gene therapies are being hailed as milestones for treating sickle cell disease and for the rapidly advancing field of gene editing, offering more targeted and effective treatments, especially for individuals with rare diseases where current treatment options are limited. The approval of these gene therapies represents a significant step forward for the sickle cell disease community, which has been historically overlooked and underfunded. The therapies are pitched as one-time treatments, with prices of $2.2 million for Casgevy and $3.1 million for Lyfgenia, and will be available in early 2024. The FDA will monitor patients throughout their lives for potential long-term safety risks, including cancer malignancies.