Political and Legislative
$2.8M Gene Therapy Treatment Approved by FDA for Patients with Rare Blood Disorder
The Food and Drug Administration (FDA) on Wednesday approved the first gene therapy treatment that could offer a permanent cure for patients who suffer from a rare blood disorder, which the manufacturer priced at $2.8 million per patient.
The cost would make the therapy one of the most expensive treatments of all time.
The therapy was developed by bluebird bio and will be sold under the brand name Zynteglo. It offers a treatment for beta-thalassemia, a type of inherited blood disorder that causes a reduction of normal hemoglobin, a protein in red blood cells that carries oxygen through the body.
