Two Sickle Cell Gene Therapies Get Landmark FDA Approval

Two cell-based gene therapies to treat sickle cell disease — Casgevy (exagamglogene autotemcel or exa-cel) and Lyfgenia (lovotibeglogene autotemcel or lovo-cel) — received FDA approval Friday, marking the first treatments to provide potential life-long elimination of severe disease symptoms.

Both products are made from the patient’s own blood stem cells, which are modified and then returned to the patient as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. The process starts with collection of the patient’s stem cells, followed by high-dose chemotherapy, then injection of the modified cells to replace the faulty bone marrow cells with cells that will produce healthy red blood cells.

Cost of the treatments has been drawing attention lately, with U.S. Senator Bill Cassidy (R-La.) seeking information on how the drugs are priced, paid for and allocated, as both therapies are expected to cost in excess of $2 million per patient. However, the Institute for Clinical and Evidence Review (ICER) weighed in on the sickle cell gene therapies last summer, concluding that even if priced at $2 million, they would be worth the cost.

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