Australian stem cell and regenerative medicine company, Cynata Therapeutics (ASX:CYP), has announced that the US FDA has granted Orphan Drug Designation for its 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 Cymerus platform manufacturing technology.
Orphan Drug Designation means CYP-001 is eligible for a range of incentives, including an extended period of marketing exclusivity, tax credits and FDA fee waivers.
GvHD is a complication that can occur after a bone marrow transplant or similar procedure. It occurs when the donor’s immune cells attack the recipient of the transplant.
The only approved treatment for GvHD is corticosteroid therapy, which is typically only effective in about 50 percent of patients. When GvHD fails to improve or worsens despite steroid treatment, patients are described as having steroid-resistant GvHD. The prognosis for these patients is poor, with mortality rates in excess of 90 percent.
Cynata recently announced 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. The Complete Response rate by Day 100 was 50 percent (GvHD signs/symptoms completely resolved in four out of eight patients).
The company said recruitment of the second (higher dose) cohort is currently progressing.
Dr Kilian Kelly, Cynata’s vice president of 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.”