FDA grants orphan drug designation to “universal, off the shelf” NK cell therapy product for the treatment of MM

On October 12, 2020, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to a “universal, off the shelf” NK cell therapy product (oNKord®) for the treatment of patients with multiple myeloma (MM).¹

An off the shelf approach, ensuring large-scale production from a single manufacturing run, may represent a valid alternative to the traditional approach by addressing the unmet need of treating large cohorts of patients.²

This NK cell therapy product, manufactured from fresh umbilical cord blood stem cells,² has already received FDA and European Medicines Agency (EMA) orphan drug designation for the treatment of acute myeloid leukemia (AML)¹. Promising results were observed in the first-in-human study in elderly patients with AML who were not eligible for allogeneic stem cell transplantation.³ Therefore, an ongoing phase I-IIa, open-label, multicenter trial (EudraCT Number 2019-003686-17) is evaluating the safety and efficacy of this product in patients with AML who are in complete morphologic remission.¹

A phase II trial for patients with MM is planned to start in 2021.

Read more about NK cell therapy for the treatment of MM, here.