The United States Food and Drug Administration (FDA) has granted Cynata Therapeutics Orphan Drug Designation for CYP-001 for the treatment of acute graft versus host disease (GvHD). 

CYP-001 is the lead mesenchymal stem cell (MSC) product manufactured using Cynata’s proprietary Cymerus platform manufacturing technology.

GvHD is a complication that can occur after a bone marrow transplant or similar procedure, when the donor’s immune cells (from the “graft”) attack the recipient of the transplant (the “host”). The only approved treatment for GvHD is corticosteroid therapy, which is typically only effective in about 50 percent of patients. 

Cynata recently announced very positive data from the first cohort of patients in its ongoing Phase 1 trial of CYP-001 for the treatment of steroid-resistant acute GvHD. No treatment-related serious adverse events or safety concerns have been identified to date, while among the eight participants in Cohort A (who received the lower dose level of CYP-001), overall survival at Day 100 was 87.5 percent, the Overall Response rate by Day 100 was 100 percent (all eight participants showed an improvement in the severity of GvHD by at least one grade compared to baseline) and the Complete Response rate by Day 100 was 50 percent (GvHD signs/symptoms completely resolved in four out of eight patients). Recruitment of the second (higher dose) cohort is currently progressing well.

Dr Kilian Kelly, Cynata’s vice president, Product Development, said, “We are delighted that the FDA has seen fit to grant Orphan Drug Designation to CYP-001, in recognition of the potential of this product to address the substantial unmet need associated with GvHD. This follows on from our successful pre-IND meeting with the FDA last year, and we look forward to building our productive relationship with the FDA over the coming years.”