The FDA on Jan. 16 approved Casgevy to be used as a treatment for beta-thalassemia. The move comes just over a month after the nation's first medicine based on CRISPR gene-editing technology was approved to treat patients with sickle cell disease.
Casgevy was approved to treat patients 12 and older with beta-thalassemia, an inherited blood disorder that affects the ability to produce hemoglobin. Patients with the condition often require frequent blood transfusions.
"Today's approval is an important step in the advancement of an additional treatment option for individuals with beta-thalassemia, a debilitating disease that places individuals at risk of many serious health problems," Nicole Verdun, MD, director of the office of therapeutic products at the FDA's center for biologics evaluation and research, said in a news release.
The treatment involves genetically modifying a patient's stem cells using the CRISPR technology and transplanting the modified cells back into the patient. The modified cells then attach and multiply within the bone marrow and increase the production of hemoglobin. Mouth sores, febrile neutropenia and decreased appetite are among the most common side effects, according to the FDA.
The list price for the treatment, made by Vertex Pharmaceuticals, is $2.2 million. It is a one-time therapy and requires administration through authorized treatment centers with experience in stem cell transplantation.