Biologics

First Sickel Cell Gene Therapies Approved

FDA has approved the first two cell-based gene therapies for treating sickle cell disease in patients 12 years and older — Vertex Pharmaceuticals’ Casgevy and Bluebird Bio’s Lyfgenia.

Casgevy is the first FDA-approved treatment to use the novel genome editing technology CRISPR/Cas9, “signaling an innovative advancement in the field of gene therapy,” the agency says in a release. “CRISPR/Cas9 can be directed to cut DNA in targeted areas, enabling the ability to accurately edit (remove, add, or replace) DNA where it was cut,” it says. “The modified blood stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery. In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.”

Lyfgenia uses a lentiviral vector as a gene delivery vehicle for genetic modification. “With Lyfgenia, the patient's blood stem cells are genetically modified to produce HbAT87Q, a gene-therapy derived hemoglobin that functions similarly to hemoglobin A, which is the normal adult hemoglobin produced in persons not affected by sickle cell disease,” FDA says. “Red blood cells containing HbAT87Q have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the patient.”

Casgevy’s approval was based on data from an ongoing single-arm trial in adult and adolescent patients with SCD, FDA says, adding that the primary efficacy outcome was freedom from severe vaso-occlusive events (VOC) for at least 12 consecutive months during the 24-month follow-up period. “Of the 31 patients with sufficient follow-up time to be evaluable, 29 (93.5%) achieved this outcome,” it says. “All treated patients achieved successful engraftment with no patients experiencing graft failure or graft rejection.”

Lyfgenia’s approval was based on data from a single-arm, 24-month study. “Effectiveness was evaluated based on complete resolution of VOEs (VOE-CR) between 6 and 18 months after infusion with Lyfgenia,” the agemcy says. “Twenty-eight (88%) of 32 patients achieved VOE-CR during this time period.”

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