EU/3/22/2667 - orphan designation for treatment of Wolfram syndrome

liraglutide
OrphanHuman

Overview

This medicine was designated as an orphan medicine for the treatment of Wolfram syndrome in the European Union on 18 July 2022.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

Most patients with Wolfram syndrome (WS) lack a protein called wolframin which plays a crucial role in the function of the pancreas and nervous system. This results in hyperglycaemia (high level of blood sugar) due to insulin shortage (diabetes mellitus) and progressive vision loss. The glucagon-like peptide-1 (GLP-1) is a gut-derived hormones which have been shown to increase insulin secretion and sensitivity, decrease inappropriate secretion of glucagon (an hormone involved in controlling blood sugar levels), delay emptying of the stomach and promote the sensation of food in the stomach (postprandial satiety). Liraglutide is an analog of human GLP-1 that binds to and activates the GLP-1 receptor in the peripheral and central nervous system, pancreas, intestine, stomach, kidney, and heart. It is expected that the administration of liraglutide before the onset of WS symptoms would offers protection against disease progression by postponing the development of glucose intolerance and reducing neuroinflammation.

Based on description provided by sponsor

At the time of submission of the application for orphan designation:

  • The effects of the medicine had been evaluated in experimental models
  • No clinical trials with the medicine in patients with Wolfram syndrome had been started.

The medicine is already authorised in the EU under the trade name Victoza for the treatment of type II diabetes, and under the trade name Saxenda for the treatment of obesity.

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

  • scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;
  • market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

  • it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or, if such a method exists, the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

Key facts

Active substance
liraglutide
Intended use
Treatment of Wolfram syndrome
Orphan designation status
Positive
EU designation number
EU/3/22/2667
Date of designation
Sponsor

Pietro Maffei

Department of Medicine (DIMED)

Via Giustiniani 2

35128 Padova

Italia

E-mail: pietro.maffei@unipd.it

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:

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