Orgenesis has received Orphan Drug Designation for a potential autologous insulin producing cell therapy to treat severe hypoglycemia-prone diabetes resulting from total pancreatectomy.
With the designation, Orgenesis hopes to expedite its Autologous Insulin Producing (AIP) cells as a cell replacement therapy for the treatment of severe hypoglycemia-prone diabetes resulting from total pancreatectomy due to chronic pancreatitis.
“The therapy is based on taking a small amount of liver cells from the patient. These are propagated to the desired amount of cells for transplantation back into the liver following reprogramming into insulin producing cells,” Vered Caplan, CEO of Orgenesis, told Bioprocess Insider.
“As with many cell-based therapies, one of the major challenges is producing the product and industrializing the process in order to make it a viable therapy. Producing the required amount of liver cells is far from a trivial task.”
Orgenesis has invested in developing the industrial capability to achieve this, she continued. “This therapeutic program is still in preclinical stage. However, Orgenesis has recently received approval to collect biopsies and build the cell banks that are the basis for this therapy.”
Cell therapy for diabetes
For severe hypoglycaemia-prone diabetes resulting from total pancreatectomy, Orgenesis is aiming to provide a one-off treatment for patients.
There is a treatment available for patients undergoing complete pancreatectomy in which their own islets are harvested from the removed pancreas and transplanted back into their own liver, but according to Caplan this is not always possible.
“In the cases where there has been availability of the autologous cells there have been promising long term results. In any case the concept is to build a biobank of cells for each patient so if there is need to replenish the cells in the future there is an available source.”
When asked if cell therapies could be used to treat patients with far less rare type 1 or 2 diabetes going forward, Caplan said Orgenesis is actively working on such applications with financial support from the Walloon Region, Belgium, along with grants from the Israeli and Korean governments and the Maryland stem cell foundation.
Allogeneic diabetes cell therapies
Approaches so far have centered on autologous cell therapies, but there have been several attempts to develop an allogeneic approach for providing insulin producing cells, Caplan said.
“The challenge in such an approach that the transplanted cells must be protected from the immune system by some type of device,” she told us.
“Insulin production requires a lot of energy from the cell and a high supply of oxygen which tends to be a challenging task when combined with the defensive barrier from the immune system. Though there has been some success in this approach, it will most likely require short term cell replacement since it is challenging to keep the cell alive in confined conditions.”
Orphan Drug Designation
The US Food and Drug Administration (FDA) Orphan Drug Designation aims to speed up development of drugs targeted at treating diseases that affect fewer than 200,000 people in the US.
“Research shows that compared with standard drugs, orphan drugs typically require shorter clinical development times, spend fewer months in regulatory review and are more likely to be approved by the FDA,” said Caplan.
A report by Premier Research adds: “The expedited development pathway and higher likelihood of FDA approval highlight the impact firms can have in the area of orphan drugs and rare diseases. Productive partnerships and proper, proactive regulatory planning can significantly reduce development time and budget, while increasing development success.”
Earlier this year, the FDA released draft guidelines for industry on the common issues in the development of rare disease drugs.
Read more: Making rare disease successes more common