Overview

On 12 December 2016, orphan designation (EU/3/16/1800) was granted by the European Commission to Alan Boyd Consultants Ltd, United Kingdom, for dantrolene sodium for the treatment of Wolfram syndrome.

The sponsorship was transferred to Boyd Consultants Limited, Ireland, in March 2019.

The sponsor’s address was updated in September 2021.

Wolfram syndrome is a group of inherited disorders that usually appear in childhood and cause diabetes, optic atrophy (when the optic nerve that connects the eye to the brain wastes away) leading to loss of vision, deafness and often 'diabetes insipidus', a condition in which the kidneys cannot retain water, resulting in excessive amounts of urine. Other complications that affect the nervous system may also occur, such as problems with balance and coordination, loss of taste and smell, breathing problems, fits and intellectual impairment.

Wolfram syndrome is a long-term debilitating condition that may be life threatening because of its complications, such as respiratory failure caused by the brain's inability to control breathing.

At the time of designation, Wolfram syndrome affected not more than 0.2 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 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 513,700,000 (Eurostat 2016).

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of Wolfram syndrome. Treatment was supportive and aimed at managing the complications of the condition, and included medicines for diabetes, to prevent fits or to treat other problems with the brain and nerves, as well as hearing aids.

Most patients with Wolfram syndrome lack a protein called wolframin, which is thought to play a role in the function of a structure called the endoplasmic reticulum inside cells, particularly in cells of the pancreas and nervous system. This lack causes the cells to become 'stressed' and die, leading to the various signs and symptoms of the disease.

Dantrolene sodium is a medicine used for many years as a muscle relaxant that acts by regulating the flow of calcium in and out of cells. In patients with Wolfram syndrome, controlling the calcium flow in this way is thought to stabilise the endoplasmic reticulum. This is expected to prevent cells from dying, therefore slowing down the progression of the disease.

The effects of dantrolene sodium 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 Wolfram syndrome had been started.

At the time of submission, dantrolene was not authorised anywhere in the EU for Wolfram syndrome 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 4 November 2016 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.

EU/3/16/1800: Public summary of opinion on orphan designation: Dantrolene sodium for the treatment of Wolfram syndrome

AdoptedReference Number: EMA/756214/2016

English (EN) (112.23 KB - PDF)

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Key facts

Active substance
Dantrolene sodium
Intended use
Treatment of Wolfram syndrome
Orphan designation status
Positive
EU designation number
EU/3/16/1800
Date of designation
Sponsor

Boyd Consultants Limited

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:

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