EU/3/18/2091 - orphan designation for treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome

Glucagon

Overview

On 19 November 2018, orphan designation (EU/3/18/2091) was granted by the European Commission to Pharma Gateway AB, Sweden, for glucagon for the treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome.

What is 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.

What is the estimated number of patients affected by the condition?

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).

 

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* 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 517,400,000 (Eurostat 2018).

What treatments are available?

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.

How is this medicine expected to work?

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.

What is the stage of development of this medicine?

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.

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.

EU/3/18/2091: Public summary of opinion on orphan designation: Glucagon for the treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome

AdoptedReference Number: EMA/769575/2018

Key facts

• Active substance:
  Glucagon
• Intended use:
  Treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2091
• Date of designation:
  19/11/2018
• Sponsor:

  S-Cubed Pharmaceutical Services ApS; Baneledet 2; 3600 Frederikssund; Hovedstaden; Denmark; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.