FDA Grants Orphan Drug Designation to Multiple Myeloma Treatment

oNKord is a natural killer cellular immunotherapy product, meaning it can play an important role in controlling and curing both solid and hematological malignancies.

Glycostem Therapeutics has received orphan drug designation for oNKord, an investigational treatment for patients with multiple myeloma. The designation provides incentives that include 7 years of market exclusivity and FDA guidance on the development process, helping accelerate the process toward market access.1

oNKord is a natural killer (NK) cellular immunotherapy product, meaning it can play an important role in controlling and curing both solid and hematological malignancies.2 According to the company, NK cells can be used alone or as adjunctive therapy, thus widening the range of treatment options.2

A study of oNKord has already been conducted in elderly patients with acute myeloid leukemia who were not eligible for allogeneic stem cell transplantation. Ten patients were treated in a phase 1 setting with an excellent safety profile and strong indication of clinical efficacy, according to Glycostem. Year 1 data from the study showed 80% survival compared with population data showing 35% for patients with no intervention.2

Multiple myeloma is the second most common blood cancer, accounting for 15% of blood cancers and 2% of all cancers. It affects more than 130,000 patients in the United States, which is still well below the 200,000-patient limit for orphan drug designation.1 A phase 2 trial for patients with multiple myeloma is expected to begin in 2021.1

REFERENCES

  • FDA grants Glycostem’s oNKord Orphan Drug Designation for Multiple Myeloma [news release]. PR Newswire; October 12, 2020. https://www.prnewswire.com/news-releases/fda-grants-glycostems-onkord-orphan-drug-designation-for-multiple-myeloma-301150019.html. Accessed October 16, 2020.
  • Glycostem, oNKord. https://glycostem.com/onkord. Accessed October 16, 2020.