FDA Grants Natural Killer Cell Therapy, CYNK-001, Fast Track Designation for Acute Myeloid Leukemia


CYNK-001 was recently granted fast track designation by the FDA for the treatment of patients with acute myeloid leukemia.

The FDA granted fast track designation to CYNK-001, a cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy that is not genetically modified, for the treatment of patients with acute myeloid leukemia (AML), according to a press release from the biotechnical company developing the agent, Celularity Inc.

CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy that incorporates CD56-positive and CD3-negative NK cells expanded from human placental CD34-positive cells and is being developed as one of Celularity’s placental-derived allogeneic cell therapies. The therapy is being developed for the treatment of hematologic malignancies, solid tumors, and infectious diseases.

“We believe that the unique properties of our cell source, including the ability to proliferate and maintain activity, could be the key to improving response rates and durability for patients,” Robert Hariri, MD, PhD, founder, chairperson, and chief executive officer of Celularity, said in a press release. “We are pleased to receive this fast-track designation from the FDA for AML supporting continued development of our placental-derived NK cell platform. CYNK-001 previously received orphan drug designation for malignant gliomas and fast track designation for glioblastoma multiforme.”

An investigational new drug application for the NK cell therapy was previously cleared by the FDA for adults who have contracted COVID-19. The previous clearance for patients with COVID-19 led to the announcement of a phase 1/2 clinical trial investigating CYNK-001 in collaboration with Sorrento Therapeutics.

The therapy is currently being assessed as part of the phase 1 CYNK001AML01 study (NCT04310592) in adult patients with AML. The study has an estimated enrollment of 56 patients who will receive an infusion of CYNK-001 at 1 of 3 dose levels. Patients will also receive a combination of cyclophosphamide and fludarabine before the experimental infusion on days 0, 7, and 14. Investigators hope to establish dose-limiting toxicities, the maximum tolerated dose, and safety profile, with key secondary outcomes including minimal residual disease, progression-free survival, overall survival, and overall response rate, among others.

CYNK-001 is not approved for any use by the FDA or any regulatory authority; its safety and efficacy data are not yet established.

“The majority of patients with AML continue to have poor long-term outcomes, particularly those who suffer relapse or have measurable residual disease, necessitating development of novel therapies, including CYNK-001,” Andrew Pecora, MD, president of Celularity, concluded.


Celularity receives fast track designation from U.S. FDA for its NK cell therapy CYNK-001 in development for the treatment of AML. News release. Celularity. December 27, 2021. Accessed January 3, 2022. https://tinyurl.com/4njx2pac

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