FDA Approves bluebird bio Gene Therapy for Sickle Cell Disease

bluebird bio has announced FDA approval for the gene therapy Lyfgenia for the treatment of sickle cell disease. The therapy has been approved for patients ages 12 and older who suffer from debilitating pain crises due to the condition. The FDA action is the second such approval in as many days, the first belonging to the Vertex/CRISPR gene therapy known as Casgevy.

Similar to Casgevy, Lyfgenia is a one-time gene therapy that is custom-designed to treat the underlying cause of sickle cell disease. Lyfgenia works by permanently inserting a modified gene into the DNA of the patients’ own hematopoietic (blood) stem cells, enabling production of adult hemoglobin with anti-sickling properties.

“Lyfgenia has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating vaso-occlusive events. This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader,” said Andrew Obenshain, chief executive officer, bluebird bio.

Three patients died during Lyfgenia clinical trials; one from sudden cardiac death due to underlying disease and two from acute myeloid leukemia who were treated with an earlier version of the therapy using a different manufacturing process and transplant procedure. The latter deaths prompted the FDA to impose a black box “hematological malignancy” warning on the instructions for use.

The Phase 1/2 study of Lyfgenia is ongoing with enrollment and treatment complete; and the Phase 3 study is ongoing. bluebird bio is also conducting a long-term safety and efficacy follow-up study for patients who have been treated with Lyfgenia in bluebird bio-sponsored clinical studies. (Source: bluebird bio Website, 08 December, 2023 and other sources)