FDA Grants Israel’s Orgenesis Orphan Drug Designation for Autologous Insulin Producing Cells

Jun 19, 2019 | Autologous Insulin Producing Cells, Diabetes, Insulin, Total Pancreatectomy

Autologous Insulin Producing (AIP) cells

Orgenesis Inc. (ORGS), a developer and service provider of advanced cell therapies, announced the 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 (CP). The technology is exclusively licensed by Orgenesis Ltd. (Orgenesis wholly-owned Israeli subsidiary) from Tel Hashomer Medical Research Infrastructure and Services Ltd. (THM) in Israel. The technology is based on the work of Professor Sarah Feber, the company’s Chief Scientific Officer and a researcher at THM. This action is expected to reduce time and cost of clinical development.

The incidence of diabetes following TP is 100%, resulting in immediate and lifelong insulin-dependence with loss of both endogenous insulin secretion and that of the counter-regulatory hormone, 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.

The company’s patented transdifferentiation (or cell reprogramming) process involves the conversion of one adult tissue or cell into another type of cell, with its distinct phenotype and function. Orgenesis 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 the company’s proprietary transdifferentiation process and returned to the patient’s liver via a transfusion. The goal is to provide a “practical cure” for various types of insulin-dependent diabetes, thus hopefully providing long-term insulin independence without the need for concomitant immunosuppressive therapy.

The FDA’s Orphan Drug Designation Program provides orphan status to drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the United States. Orphan designation qualifies the sponsor of the drug for various development incentives, including eligibility for seven years of market exclusivity upon regulatory approval, exemption from FDA application fees, tax credits for qualified clinical trials and other potential assistance in the drug development process.

About Orgenesis

Orgenesis is a biotechnology company specializing in the development, manufacturing and provision of technologies and services in the cell and gene therapy industry.  The Company operates through two platforms, a POCare cell therapy platform (“PT”) and a Contract Development and Manufacturing Organization (“CDMO”) platform conducted through its subsidiary, Masthercell Global Inc. (“Masthercell Global”).  Through its PT business, the Company’s aim is to further the development of Advanced Therapy Medicinal Products (“ATMPs”) through collaborations and in-licensing with other pre-clinical and clinical-stage biopharmaceutical companies and research and healthcare institutes to bring such ATMPs to patients.  The Company out-licenses these ATMPs through regional partners to whom it also provides regulatory, pre-clinical and training services to support their activity in order to reach patients in a point-of-care hospital setting.  Through the company’s CDMO platform, it is focused on providing contract manufacturing and development services for biopharmaceutical companies.  The CDMO platform operates through Masthercell Global, which currently consists of MaSTherCell S.A. in Belgium, Atvio Biotech Ltd. in Israel and subsidiaries in South Korea and in the United States, each having unique know-how and expertise for manufacturing in a multitude of cell types.  Additional information is available at: www.orgenesis.com.


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