EU/3/14/1342 - orphan designation for treatment of congenital hyperinsulinism

Glucagon

Overview

On 15 October 2014, orphan designation (EU/3/14/1342) was granted by the European Commission to S-cubed Limited, United Kingdom, for glucagon for the treatment of congenital hyperinsulinism.

What is congenital hyperinsulinism?

Congenital hyperinsulinism is an inherited disorder caused by high levels of insulin, a hormone that helps control blood glucose (sugar) levels. Insulin lowers blood glucose levels by driving glucose into the cells of the body. In hyperinsulinism, more insulin is produced than is needed which results in hypoglycaemia (low blood glucose levels). The severity of congenital hyperinsulinism varies among patients, with some patients already developing episodes of hypoglycaemia shortly after birth. Repeated episodes of hypoglycaemia increase the risk of serious complications such as seizures (fits), mental disability, breathing difficulties and coma.

Congenital hyperinsulinism is a long-term debilitating and life-threatening disease since glucose is essential for brain cells to function, and severely low blood sugar levels can cause permanent brain injury or death.

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

At the time of designation, congenital hyperinsulinism affected not more than 2 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 102,000 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 511,100,000 (Eurostat 2014).

What treatments are available?

At the time of designation, no satisfactory methods of treatment were authorised in the EU for congenital hyperinsulinism. The main treatment consisted of infusions of glucose to maintain normal glucose levels. Medicines such as diazoxide and octreotide were used to reduce insulin secretion, and glucagon injections were used to release glucose from the liver and thereby increase blood glucose levels. However, existing glucagon formulations could not be used regularly over long periods because of poor stability.

How is this medicine expected to work?

This medicine is a synthetic form of glucagon, a hormone naturally secreted by the pancreas that counteracts the effects of insulin by raising blood glucose levels. It is expected to be given under the skin using a special infusion pump. This is expected to help increase blood sugar levels, thereby improving the symptoms of the disease. The way the medicine is formulated is expected to make it suitable to be given regularly for long periods.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of glucagon in experimental models was ongoing.

At the time of submission, clinical trials with glucagon in patients with congenital hyperinsulinism had not started.

At the time of submission, glucagon was authorised in the EU and the United States for the treatment of severe hypoglycaemia.

At the time of submission, glucagon was not authorised anywhere in the EU for the treatment of congenital hyperinsulinism. Orphan designation for glucagon in the treatment of congenital hyperinsulinism had been previously granted in the EU.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 September 2014 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/3/14/1342: Public summary of opinion on orphan designation: Glucagon for the treatment of congenital hyperinsulinism

Adopted Reference Number: EMA/COMP/546576/2014

Key facts

• Active substance:
  Glucagon
• Intended use:
  Treatment of congenital hyperinsulinism
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/14/1342
• Date of designation:
  08/10/2009
• Sponsor:

  S-Cubed Pharmaceutical Services ApS;

Review of designation

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