EU/3/12/1081 - orphan designation for treatment of very-long-chain 3-hydroxyacyl-CoA-dehydrogenase deficiency

Triheptanoin
OrphanHuman

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in February 2016 on request of the Sponsor.

On 6 December 2012, orphan designation (EU/3/12/1081) was granted by the European Commission to B. Braun Melsungen AG, Germany, for triheptanoin for the treatment of very-long-chain 3-hydroxyacyl-CoA-dehydrogenase deficiency.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

Very-long-chain 3-hydroxyacyl-CoA-dehydrogenase (VLCAD) deficiency is an inherited disease caused by the lack of an enzyme called VLCAD. VLCAD is one of the enzymes needed by the mitochondria (the energy-producing components within cells) to break down certain long fatty acids in order to generate energy. If this enzyme is not present, cells cannot function normally, causing a wide range of signs and symptoms including tiredness, hypoglycaemia (low blood sugar levels), muscle wasting and damage to the heart.

The condition is chronically debilitating and life-threatening, particularly as it causes damage to the heart.

At the time of designation, VLCAD deficiency affected not more than 0.32 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 16,000 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).


*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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 509,000,000 (Eurostat 2012).

At the time of orphan designation, no satisfactory method had been authorised in the European Union for the treatment of VLCAD deficiency. Treatment of patients primarily involved restriction of dietary fat to less than 30% of the total calories and the substitution of long-chain fatty acids with medium-chain fatty acids. However, these dietary regimens were of unproven value or only partially successful.

Triheptanoin is a synthetic (artificially produced) fat, which is broken down in the liver into substances that can be used to generate energy without the need for VLCAD. By bypassing the need for VLCAD, this medicine is expected to restore the normal energy generation and ultimately improve the overall outcome of the patients.

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

At the time of submission of the application for orphan designation, no clinical trials with triheptanoin in patients with VLCAD deficiency had been started.

At the time of submission, triheptanoin was not authorised anywhere in the EU for VLCAD deficiency. Orphan designation of triheptanoin had been granted in the United States for fatty-acid-oxidation disorders.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 November 2012 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
Triheptanoin
Intended use
Treatment of very-long-chain 3-hydroxyacyl-CoA-dehydrogenase deficiency
Orphan designation status
Withdrawn
EU designation number
EU/3/12/1081
Date of designation
Sponsor

B. Braun Melsungen AG
Carl-Braun-Strasse 1
34212 Melsungen
Germany
Tel. +49 56 61 710
Fax +49 56 61 71 2950

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:

  • 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.

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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