FDA approves two gene therapies for sickle cell, bringing hope to thousands with the disease

(CNN) ­– The U.S. Food and Drug Administration approved two gene-based treatments for sickle cell disease Friday, including the first therapy that uses the gene-editing technique CRISPR, opening a new era of treatments for genetic conditions. The medicines, called Casgevy and Lyfgenia, are potential cures for people with sickle cell, a debilitating and life-shortening inherited red blood cell disorder that disproportionately affects African Americans. Casgevy is the CRISPR-based treatment, made by Vertex Pharmaceuticals and Crispr Therapeutics; Lyfgenia, made by bluebird bio, uses an older gene therapy approach. Both were cleared for people 12 and older with histories of vaso-occlusive crises, painful events caused by the disease. “Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need,” said Dr. Nicole Verdun, director of the FDA’s Office of Therapeutic Products within its Center for Biologics Evaluation and Research. “We are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today.” Related Stories Casgevy will cost $2.2 million for the one-time treatment, Vertex said in a regulatory filing, while Lyfgenia will cost $3.1 million, bluebird said in a news release. A drug pricing analysis...