Pluristem Therapeutics Inc., a leading developer of placenta-based cell therapy products, has announced that the U.S. Food and Drug Administration has granted the Company's PLX-PAD cells Orphan Drug Designation in the treatment of severe preeclampsia. Preeclampsia is among the most common medical complications of pregnancy and a leading cause of premature births, stillbirths and neonatal and maternal deaths. There is no cure except delivery. Due to high risks to the mother, women diagnosed with severe preeclampsia are usually delivered promptly, even if the baby will be born prematurely and may suffer permanent disabilities as a result. Severe preeclampsia occurs in approximately 1% of pregnancies in Western countries.
Benefits of Orphan Drug Designation for PLX-PAD cells include close guidance that may accelerate time to marketing approval, orphan drug grants, tax credits, and a 7-year market exclusivity upon marketing approval. It is estimated by different sources that preeclampsia costs the global health care system about $3 billion annually.
"Attainment of Orphan Drug Designation for our cells in severe preeclampsia exemplifies our global strategy of bringing cell therapies to patients through accelerated approval pathways," stated Pluristem Chairman and CEO Zami Aberman. "We are encouraged by the US FDA designation that demonstrates Pluristem's commitment to the program and the potential promise it holds to address a serious, unmet medical need faced by pregnant women every year."
PLX-PAD cells improved several parameters of preeclampsia in animal models in a study conducted in collaboration with Brett Mitchel PhD, Associate Professor of Internal Medicine at the Cardiovascular Research Institute (CVRI) of the Texas A&M College of Medicine. In a different animal study conducted by Charles River Laboratories, PLX-PAD cells were demonstrated to be safe to be safe for both the mother and fetus. As previously requested by the FDA, study in additional animal model (over expression of sFLT-1) to confirm the efficacy of PLX-PAD is on-going to test the efficacy of PLX-PAD in an additional therapeutic pathway. Data is expected in H1 of 2016.