FDA approves CAR-T therapy for multiple myeloma

The FDA gas approved a therapy developed by J&J and its partner Legend Biotech, to treat relapsed or refractory multiple myeloma (RRMM), a form of white blood cell cancer. The approval could ease strain on limited supplies of potentially lifesaving cancer therapies.

The approval marks Legend’s first approved product in the US. The treatment, CARVYKTI ™ /Cilta-cell (ciltacabtagene autoleucel), is a CAR-T cell therapy, a group of therapies which work by genetically altering a patient’s T-cells so that they target specific proteins in cancer cells. In this way the immune cells from a patient’s own body are engineered to fight their cancer. The first treatment of this kind for multiple myeloma was approved last year.

Multiple myeloma is an incurable blood cancer, in which most patients face poor prognoses after experiencing disease progression following treatment.

“Multiple myeloma remains an incurable disease with heavily pretreated patients facing poor prognoses with limited treatment options,” said Ying Huang, PhD, CEO and CFO of Legend Biotech. “Today’s approval of CARVYKTI is a pivotal moment for Legend Biotech because it is our first-ever marketing approval, but what really excites us is the drug’s potential to become an impactful therapy option for patients in need of long, treatment-free intervals. This is the first of many cell therapies we plan to bring to patients as we continue advancing our pipeline across disease states.”

The approval is based on data from the pivotal CARTITUDE-1 study. One-time treatment with ciltacabtagene autoleucel resulted in deep and durable responses, with 98% of patients with RRMM responding to therapy.

Alongside this, 78% of the patients achieving this level of response experienced a stringent complete response (sCR), a measure in which a physician is unable to observe any signs or symptoms of disease via imaging or other tests after treatment.

Ana Ovey