EU/3/16/1750 - orphan designation for treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome

Exendin (9-39)

Overview

On 14 October 2016, orphan designation (EU/3/16/1750) was granted by the European Commission to Eiger Biopharmaceuticals Europe Ltd, United Kingdom, for exendin (9-39) for the treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome.

The sponsorship was transferred to Eigerbio Europe Limited, Ireland, in August 2019.

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 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 190,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of orphan designation there was no satisfactory treatment authorised in the EU for NIPHS. Patients were sometimes treated with medicines such as diazoxide that help to reduce insulin secretion or given surgery to reduce the size of the pancreas and so the numbers of insulin-producing cells.

How is this medicine expected to work?

Release of insulin from the insulin-producing cells in the pancreas is triggered by a natural hormone, glucagon-like peptide-1 (GLP-1), when it attaches to receptors on these cells. Exendin (9-39) is able to attach to and block the GLP-1 receptors. This prevents GLP-1 from triggering insulin release, resulting in more normal blood sugar levels in patients with NIPHS.

What is the stage of development of this medicine?

The effects of exendin (9-39) 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, exendin (9-39) was not authorised anywhere in the EU for NIPHS. Orphan designation of the medicine had been granted in the United States for treatment of congenital (inborn) hyperinsulinaemic hypoglycaemia and other causes of low blood sugar due to excess insulin secretion in adults and children.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 September 2016 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/3/16/1750: Public summary of positive opinion for orphan designation of exendin (9-39) for the treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome

AdoptedReference Number: EMA/622426/2016

Key facts

• Active substance:
  Exendin (9-39)
• Intended use:
  Treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1750
• Date of designation:
  14/10/2016
• Sponsor:

  Eigerbio Europe Limited; 1 Castlewood Avenue; Rathmines; Dublin 6, D06 H685; Ireland; Tel. [phone]; E-mail: [e-mail]