FDA grants orphan drug status to an investigational NK cell-based therapy for the treatment of multiple myeloma

On November 4, 2020, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to an investigational natural killer (NK) cell-based therapy (CellProtect) for the treatment of newly diagnosed multiple myeloma.¹

The orphan drug designation of this autologous NK cell-based product was based on the results from the first-in-human phase I/II ACP-001 clinical trial (NCT04558853).² This open-label, single-arm trial evaluated the safety and efficacy of the investigational NK cell product in 12 patients as consolidation, following high dose therapy plus autologous stem cell transplantation (auto-SCT). The product was infused in a triple escalating dose:²

• 1st infusion: 5 × 10⁶ cells/kg
• 2nd infusion: 50 × 10⁶ cells/kg
• 3rd infusion: 100 × 10⁶ cells/kg

The product showed a good safety profile, with no severe adverse events reported, and promising efficacy data: four out of six patients with measurable disease after auto-SCT improved their depth of response after NK cell infusion.²

A phase II study will evaluate this autologous NK cell-based product combined with isatuximab (an anti-CD38 monoclonal antibody) as maintenance after auto-SCT. This clinical trial is planned to begin recruitment soon (NCT04558931).²