EU/3/16/1721: Orphan designation for the prevention of graft loss in pancreatic islet transplantation

Pancreatic islets are clusters of cells (beta cells) in the pancreas which produce insulin. Pancreatic islet transplantation can be used to treat patients with type 1 diabetes that is difficult to control.

Graft loss occurs when the recipient's body rejects the transplanted pancreatic islets (the 'graft'). It is caused by the patient's immune system (the body's natural defences) recognising the transplanted cells as 'foreign' and reacting against them. This results in inflammation and damage to the graft.

Graft rejection in pancreatic islet transplantation is debilitating in the long term because it can lead to failure to cure type 1 diabetes.

At the time of designation, patients at risk of graft loss in pancreatic islet transplantation were less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 513,700,000 (Eurostat 2016).

At the time of designation, no satisfactory methods were authorised in the EU for the prevention of graft loss in pancreatic islet transplantation.

The medicine is a short chain of amino acids that is able to attach to and activate a receptor in the body called the innate repair receptor (IRR). The IRR is produced at high levels in cells, including pancreatic islet cells, when there is tissue damage or inflammation. When the medicine attaches to the receptor, it stimulates repair processes in the cells and tissues which help to control inflammation and apoptosis (programmed cell death). This is expected to reduce the loss of islet cells from the graft following transplantation.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients who had undergone pancreatic islet transplantation had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for prevention of graft loss in pancreatic islet transplantation. Orphan designation of the medicine had been granted in the USA for prevention of delayed graft function following kidney transplant.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 July 2016 recommending the granting of this designation.

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.