Novel Cell Therapy Product for Bone Marrow Failure Syndromes Receives FDA Aplastic Anemia Orphan Drug Designation

The FDA has granted Orphan Drug Designation (ODD) for treatment of aplastic anemia to CK0801, a novel cell therapy product. This decision was announced in a news release from Cellenkos Inc, the biotechnology company developing CK0801.

CK0801 is an allogeneic, umbilical cord blood–derived T regulatory cell product designed by Cellenkos to promote bone marrow function and restore immune balance in patients with aplastic anemia and other bone marrow failure syndromes.

“We aim to deliver a transformative therapeutic that can reduce the burden of blood and platelet transfusions in patients with Aplastic Anemia who have failed to respond to standard-of-care treatment,” Cellenkos founder Simrit Parmar, MD, commented in the press release.¹

A phase 1 dose-escalation study of CK0801, the findings of which were published in NEJM Evidence, evaluated the product in patients with aplastic anemia, myelofibrosis, or hypoplastic myelodysplasia who had a suboptimal response to prior conventional therapies. Seventy-five percent of patients with aplastic anemia achieved partial responses, but favorable efficacy and safety results were observed across all 3 conditions in the trial.²

“In previously treated patients, CK0801 demonstrated no dose-limiting toxicity and showed evidence of efficacy, providing proof of concept for targeting inflammation as a therapy for bone marrow failure,” wrote study first author Tapan M. Kadia, MD, of the University of Texas MD Anderson Cancer Center in Houston, and colleagues.²

Cellenkos is currently in the process of filing for a registration trial to advance regulatory approval for use of CK0801 in management of transfusion-dependent aplastic anemia. This trial is projected to take place in the second half of 2025.¹

The phase 1 clinical trial was funded by Cellenkos.

References

1. Cellenkos announces US FDA Orphan Drug Designation granted to CK0801 (allogeneic cord blood derived T regulatory cell product) for treatment of aplastic anemia. News release. PR Newswire. April 14, 2025. Accessed April 15, 2025. https://www.prnewswire.com/news-releases/cellenkos-announces-us-fda-orphan-drug-designation-granted-to-ck0801-allogeneic-cord-blood-derived-t-regulatory-cell-product-for-treatment-of-aplastic-anemia-302427571.html
2. Kadia TM, Huang M, Pemmaraju N, et al. Phase 1 study of CK0801 in treatment of bone marrow failure syndromes. NEJM Evid. 2024;3(6):EVIDoa2300362. doi:10.1056/EVIDoa2300362