EU/3/17/1887 - orphan designation for treatment of congenital hyperinsulinism
Synthetic glucagon analogue modified to contain 7 amino acid substitutions
OrphanHuman
Overview
On 20 June 2017, orphan designation (EU/3/17/1887) was granted by the European Commission to Zealand Pharma A/S, for synthetic glucagon analogue modified to contain 7 amino acid substitutions for the treatment of congenital hyperinsulinism.
The sponsor’s address was updated in September 2020.
Congenital hyperinsulinism is an inherited disorder in which the body releases insulin even when it is not needed. Insulin is a hormone that helps control blood glucose (sugar) levels by driving glucose into the cells of the body. In hyperinsulinism, the increased amount of insulin causes hypoglycaemia (low blood glucose levels). The severity of congenital hyperinsulinism varies among patients and some patients develop 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 condition because of the effects of long-term hypoglycaemia on the brain, such as mental disability and seizures.
At the time of designation, congenital hyperinsulinism affected approximately 0.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 10,000 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).
*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 515,700,000 (Eurostat 2017).
At the time of designation, glucagon and glucose were used in patients with congenital hyperinsulinism to increase blood glucose levels.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with the condition. Unlike other glucagon preparations, this medicine has been modified to improve its stability making it more suitable for long-term use. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is a synthetic copy of the natural hormone glucagon that counteracts the effects of insulin by raising blood glucose levels. It does this by breaking down glycogen (the form in which glucose is stored in the liver), and by stimulating the production of glucose in the liver. The medicine is therefore expected to prevent hypoglycaemic episodes and organ damage due to congenital hyperinsulinism.
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 with congenital hyperinsulinism were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for congenital hyperinsulinism 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 12 May 2017 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.
Key facts
- Active substance
- Synthetic glucagon analogue modified to contain 7 amino acid substitutions
- Intended use
- Treatment of congenital hyperinsulinism
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1887
- Date of designation
- Sponsor
Zealand Pharma A/S
Sydmarken 11
2860 Soeborg
Denmark
E-mail: MKronborg@zealandpharma.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: