Germantown, MD—Orgenesis Inc., a developer, manufacturer and service provider of advanced cell therapies, announced today that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug designation for its autologous insulin producing (AIP) cells as a cell replacement therapy for the treatment of severe hypoglycemia-prone diabetes resulting from total pancreatectomy (TP) due to chronic pancreatitis.
“We are honored to receive Orphan Drug designation from the FDA, as this represents a major milestone for both Orgenesis and patients who have to suffer the tremendous hardships associated with total pancreatectomy. Orphan Drug designation has the potential to reduce the time and costs required to bring our AIP cell therapy to market and should help streamline the approval process,” said Vered Caplan, CEO of Orgenesis.
The AIP technology is exclusively licensed by Orgenesis Ltd., Orgenesis Inc.’s wholly owned subsidiary, from Tel Hashomer Medical Research Infrastructure and Services Ltd. in Israel (THM). It is based on the work of Professor Sarah Ferber, Orgenesis’ chief science officer and a researcher at THM.
The incidence of diabetes following TP is 100 percent, resulting in immediate and lifelong insulin-dependence with the loss of both endogenous insulin secretion and glucagon. Glycemic control after TP is notoriously difficult with conventional insulin therapy, due to complete insulin dependence and loss of glucagon-dependent counter-regulation. Patients with this condition experience both severe hyperglycemic and hypoglycemic episodes.
“This therapy is a great example of what we hope to achieve on a broader scale and for numerous indications through our POCare strategy. By collaborating with multiple clinical sites to collect and process the liver biopsies from the relevant patient populations, we are building the framework for the creation and maintenance of our liver cell banks that will be the basis for the transdifferentiation process,” continued Caplan. “Collaborations such as the NY Blood Center in the US and the Medical University of Graz in Austria will allow us to maintain liver cell banks at a level required for clinical use and will become a vital resource for clinical development.”
Orgenesis’ patented transdifferentiation process involves the conversion of one adult tissue or cell into another type of cell, with its distinct phenotype and function. The company has developed a novel therapy utilizing its transdifferentiation process and its Point of Care (POCare) liver expansion technology to transform liver cells into AIP cells. The cells are first derived from a small sample of the patient’s liver cells and expanded in a liver cell bank based on Orgenesis-unique POCare cell expansion capabilities. At the appropriate time, the cells may be converted into functional glucose-regulated AIP cells through Orgenesis’ proprietary transdifferentiation process and returned to the patient’s liver via transfusion.
“While the treatment of patients with hypoglycemia-prone diabetes resulting from total pancreatectomy is our immediate focus, we see significant opportunities ahead to expand our indications to include treatment for other causes of diabetes,” Caplan noted.
The company’s goal is to provide a practical cure for various types of insulin-dependent diabetes, hopefully providing long-term insulin independence without the need for concomitant immunosuppressive therapy.