EU/3/18/2091: Orphan designation for the treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome

Noninsulinoma pancreatogenous hypoglycaemia syndrome (NIPHS) is a disorder characterised by excess growth of cells in the pancreas (a small organ behind the stomach) that make insulin. As a result, patients with this syndrome produce too much insulin and are prone to episodes of abnormally low blood sugar (hypoglycaemia). NIPHS can occur as an inborn condition in children due to genetic changes or can develop following weight-loss surgery in adults.

NIPHS is a life-threatening and long-term debilitating condition because repeated episodes of low blood sugar can cause fits, loss of consciousness, heart problems and brain damage.

At the time of designation, NIPHS affected approximately 3.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 191,000 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).

At the time of designation, there were no treatments authorised in the EU for NIPHS. Patients often underwent surgery to remove some of the pancreas. Products such as diazoxide and octreotide were used to reduce insulin secretion. However, these medicines were not authorised for use in the condition.

The medicine is a synthetic form of the hormone glucagon, which is naturally produced by the body. It acts like natural glucagon and releases glucose from the liver, which increases blood glucose levels thereby reducing the symptoms of the disease.

The effects of glucagon have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with NIPHS were ongoing.

At the time of submission, glucagon injections were authorised for use in emergency situations to treat episodes of hypoglycaemia in diabetes when the patient is unconscious.

At the time of submission, the medicine was not authorised anywhere in the EU for NIPHS or designated as an orphan medicinal product elsewhere for this condition. 

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 October 2018 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.