EU/3/17/1879 - orphan designation for treatment of epidermolysis bullosa
Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7)
talfirastide
Orphan
Human
Overview
This medicine is now known as Angiotensin 1-7.
On 20 June 2017, orphan designation (EU/3/17/1879) was granted by the European Commission to Envigo Pharma Consulting Limited, United Kingdom, for Asp-Arg-Val-Tyr-Ile-His-Pro (also known as TXA127) for the treatment of epidermolysis bullosa.
Epidermolysis bullosa is a group of inherited diseases of the skin, in which the skin is very fragile and forms severe blisters after even minor friction (rubbing) or injury. In most cases, symptoms of epidermolysis bullosa appear from birth, but for some forms, symptoms may not occur until adulthood. The diseases are caused by mutations (changes) in the genes responsible for the production of certain proteins that make the skin strong and elastic, such as collagen or keratins.
Epidermolysis bullosa is a long-term debilitating and life-threatening condition because the severe blistering and associated scarring and deformities result in poor quality of life and may also reduce life expectancy.
At the time of designation, epidermolysis bullosa affected approximately 0.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 36,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 515,700,000 (Eurostat 2017).
At the time of designation, no satisfactory methods were authorised in the EU to treat epidermolysis bullosa. Good personal hygiene and skincare were recommended to help blisters heal, to avoid infections and to protect the skin from damage. Painkillers were also used. Surgery was sometimes necessary for complications such as deformed hands or the development of skin cancer.
The medicine is a synthetic form of angiotensin-(1-7), which is a substance naturally found in the body that reduces the development of scar tissue (fibrosis) and inflammation. How it works in the treatment of epidermolysis bullosa is not completely understood but is thought to involve reducing fibrosis in the skin by stimulating a receptor (target) on cells called the Mas receptor. The medicine also reduces inflammation. It is expected that these effects will help to reduce the symptoms of epidermolysis bullosa.
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 the medicine in patients with epidermolysis bullosa had been started.
At the time of submission, the medicine 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 12 May 2017 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
- Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7)
- talfirastide
- Intended use
- Treatment of epidermolysis bullosa
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1879
- Date of designation
- Sponsor
Eliquent Life Sciences Limited
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| August 2024 | The sponsorship was transferred from Rapport Global Strategic Services Ireland Limited to Eliquent Life Sciences Limited |
| January 2020 | The sponsorship was transferred from Envigo CRS S.A. to Rapport Global Strategic Services Ireland Limited. |
| April 2019 | The sponsorship was transferred to Envigo CRS S.A. |
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: