February 20, 2014
Pluristem Therapeutics, Inc., a leading developer of placenta-based cell therapies, today announced it is submitting its application to the United Stated Food and Drug Administration requesting the Company to be granted Orphan Drug Designation for its PLacental eXpanded (PLX-PAD) cells in the treatment of severe preeclampsia.
Orphan Drug Designation may qualify a company for several benefits under the Orphan Drug Act of 1983 (ODA), as amended. These benefits may include a 7-year period of orphan drug exclusivity upon product approval, a tax credit for certain clinical testing expenses for the orphan drug, written guidance on the non-clinical and clinical studies needed to obtain marketing approval of an orphan drug, and orphan drug grants.
“Having just recently established our Preeclampsia Steering Committee comprised of key medical opinion leaders, this Orphan Drug application submission is an important step in advancing our preeclampsia development program,” stated Pluristem Chairman and CEO Zami Aberman. “The tremendous unmet medical need makes this indication one of our top priorities. We look forward to advancing into human trials and we are hopeful that the very strong preclinical data for PLX cells will translate into similar results at the clinical level.”
Pluristem has successfully received Orphan Drug Designation from the FDA for its PLX cells in two other indications: the treatment of aplastic anemia; and the treatment of Buerger’s disease.
Preeclampsia is one of the most common medical complications of pregnancy, and one of the leading known causes of premature births, stillbirths and early neonatal and maternal deaths. If left untreated it can progress to eclampsia, the life-threatening occurrence of seizures during pregnancy. The only definitive treatment for preeclampsia is abortion or delivery. The disease occurs after the 20th week of pregnancy, and is characterized by high blood pressure and significant amounts of protein in the urine or end-organ dysfunction. According to the World Health Organization, preeclampsia occurs in approximately 6%–8% of pregnancies worldwide. It is estimated that preeclampsia costs the global health care system $3 billion annually.
See the full press release here.