EU/3/10/845 - orphan designation for treatment of epidermolysis bullosa

dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract)
OrphanHuman

Overview

On 23 February 2011, orphan designation (EU/3/10/845) was granted by the European Commission to Birken GmbH, Germany, for dry extract from birch bark (DER 0.1-0.2:1), extraction solvent n-heptane 95% (V/V) for the treatment of epidermolysis bullosa.

Following a corrigendum procedure, the active ingredient was renamed from dry extract from birch bark (DER 0.1-0.2:1), extraction solvent n-heptane 95% (V/V) to dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) in March 2021.

This medicine is now known as birch bark extract.

The medicinal product has been authorised in the EU as Filsuvez since 21 June 2022.

Epidermolysis bullosa is an inherited disease of the skin that mainly affects babies and children, in which the outer layer of the skin, the epidermis, separates from the inner layer, the dermis. This makes the skin very fragile and causes severe blistering and scarring. The disease is caused by abnormalities in the genes responsible for the production of the proteins that make the skin strong and elastic, such as collagen.

Epidermolysis bullosa is a disease that is debilitating in the long term and may be life threatening, mainly because of the severe blistering, which results in poor quality of life and reduced life expectancy.

At the time of designation, epidermolysis bullosa affected less than 0.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 25,000 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. This represents a population of 506,500,000 (Eurostat 2010).

At the time of application, no satisfactory methods were authorised in the EU to treat epidermolysis bullosa. Patients were advised to maintain a high standard of personal hygiene and skincare to help blisters heal, to avoid infections and to protect the skin from damage. Painkillers were also used.

Surgery was sometimes necessary if there were complications such as deformed hands or the development of skin cancer.

Dry extract from birch bark (DER 0.1-0.2:1), extraction solvent n-heptane 95% (V/V) is a herbal product that contains naturally-occurring substances known as triterpenes. Triterpenes are expected to have an effect on the cells in the epidermis, called keratinocytes, by speeding up the multiplication and development of these cells. When applied to wounds and blisters in the form of an ointment, its effect on the keratinocytes is expected to speed up the body's wound repair process.

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 dry extract from birch bark in patients with epidermolysis bullosa had been started.

At the time of submission, dry extract from birch bark was not authorised anywhere in the EU for epidermolysis bullosa 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 8 December 2010 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
dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract)
Intended use
Treatment of epidermolysis bullosa
Orphan designation status
Positive
EU designation number
EU/3/10/845
Date of designation
Sponsor

Chiesi Farmaceutici S.p.A.
 

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

The Committee for Orphan Medicinal Products reviewed the orphan designation of Filsuvez at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the Filsuvez : EPAR - Orphan Maintenance Assessment Report (initial authorisation).

Update history

DateUpdate
July 2024The sponsorship was transferred to Chiesi Farmaceutici S.p.A.
March 2021The sponsorship was transferred to Amryt Pharmaceuticals Designated Activity Company, Ireland
October 2019The sponsorship was transferred to Amryt Research Limited, Ireland

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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