FDA grants orphan drug designation to natural killer cell therapy for multiple myeloma
The FDA granted orphan drug designation to oNKord, an investigational natural killer cell-based therapy, for the treatment of multiple myeloma, according to the agent’s manufacturer.
oNKord (Glycostem Therapeutics) is an allogeneic natural killer cell product generated ex vivo from CD34-positive hematopoietic stem and progenitor cells from partially HLA-matched umbilical cord blood units. The therapy received FDA orphan drug designation in 2016 for adults with acute myeloid leukemia.
The phase 1, first-in-human study of oNKord for AML was conducted in the Netherlands among 10 older patients ineligible for allogeneic stem cell transplantation. Treatment resulted in no dose-limiting toxicities or cases of graft-versus-host disease, and patients demonstrated a reduction of residual disease and prolonged PFS and OS.
Patients with AML will receive oNKord as part of Glycostem’s ongoing phase 1/phase 2a (pivotal) clinical trial. A phase 2 trial for patients with multiple myeloma is set to start in 2021.
"Since 2012, we have been pioneers in the field of developing and manufacturing off-the-shelf natural killer cell therapy products for cancer treatment. In 2020, we are entering a new and exciting phase," Troels Jordansen, CEO of Glycostem, said in a company-issued press release.
"After receiving FDA and [European Medicines Agency orphan drug designation] for AML, the FDA has also granted us this designation for multiple myeloma,” he added. “This allows us to accelerate oNKord's access to the U.S. market and our ultimate ambition: curing cancer."
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.