Photo by Chad McNeeley
Hematopoietic stem cell transplant preparation
The US Food and Drug Administration (FDA) has granted orphan drug designation to ApoGraft™ as prophylaxis for acute and chronic graft-versus-host disease (GVHD) in transplant recipients.
ApoGraft is a mobilized peripheral blood cell product collected via apheresis from a matched, related donor. The product is exposed to the apoptotic mediator Fas ligand prior to transplantation.
ApoGraft was designed to eliminate immune responses after transplantation of foreign cells and tissues.
ApoGraft is being developed by Cellect Biotechnology Ltd.
The company is testing ApoGraft as acute GVHD prophylaxis in a phase 1/2 trial.
The trial is currently enrolling patients with hemato-oncology disorders who are eligible for allogeneic, HLA-matched hematopoietic stem cell transplant (HSCT).
The study is expected to have 4 cohorts, each consisting of 3 patients.
The difference between the cohorts is the amount of apoptotic mediator Fas ligand (APO010) to which the graft is exposed during incubation prior to ApoGraft transplantation and HSCT:
- 10 ng/mL APO010 in Cohort 1
- 25 ng/mL APO010 in Cohort 2
- 50 ng/mL APO010 in Cohort 3
- 100 ng/mL APO010 in Cohort 4.
The study is expected to progress from one cohort to the next based on an independent data safety monitoring board review and analysis of safety data.
About orphan designation
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
