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Orphan designation

On 15 January 2016, orphan designation (EU/3/15/1589) was granted by the European Commission to AMMTeK, France, for glibenclamide for the treatment of neonatal diabetes.

The sponsorship was transferred to Pharma Services, France, in September 2016 and back to AMMTeK, France, in February 2017.

Glibenclamide has been authorised in the EU as Amglidia since 24 May 2018.

What is neonatal diabetes?

Neonatal diabetes is a form of diabetes that is diagnosed in the first 6 months of life. This condition is distinct from the more common type 1 diabetes (which occurs later).

Signs and symptoms are those that typically occur with poor blood sugar control including thirst, frequent urination, dehydration and, in severe cases, a harmful build-up of acid (ketoacidosis). Infants with neonatal diabetes may also suffer nervous system disorders which may cause muscle weakness and convulsions (fits).

Neonatal diabetes is life threatening and debilitating in the long term because of the symptoms caused by high blood sugar levels and the risk of ketoacidosis.

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

At the time of designation, neonatal diabetes affected less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 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 512,900,000 (Eurostat 2015).

What treatments are available?

At the time of the orphan designation, insulin was authorised in the EU for use in neonatal diabetes.

The sponsor has provided sufficient information to show that glibenclamide might be of significant benefit for patients with neonatal diabetes because early studies indicate that patients treated with glibenclamide may not need to be treated with insulin or may need less insulin. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Glibenclamide is authorised for treating type 2 diabetes. It works on insulin-producing cells in the pancreas by attaching to a KATP channel, which controls the release of insulin. In many newborn babies with neonatal diabetes, the cells in the pancreas produce insulin but they are not able to release it into the blood. The lack of insulin in the blood causes symptoms of diabetes. Glibenclamide's effect on the KATP channel restores the cells' ability to release insulin into the blood. Glibenclamide is also thought to make the rest of the body respond more effectively to insulin. These effects are expected to reduce the symptoms of neonatal diabetes.

What is the stage of development of this medicine?

The effects of glibenclamide 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 neonatal diabetes were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for neonatal diabetes 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 November 2015 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.

Key facts

Product details for <p>Glibenclamide for the treatment of neonatal diabetes</p>
Active substanceGlibenclamide for the treatment of neonatal diabetes
Medicine Name
Disease/conditionTreatment of neonatal diabetes
Date of decision15/01/2016
Orphan decision numberEU/3/15/1589

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Amglidia at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the orphan medicine assessment report.

Related information

Sponsor’s contact details

15 rue Béranger
75003 Paris
Tel. +33 158 281 680
Fax +33 1 58 28 16 90

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.