EU/3/17/1951 - orphan designation for treatment of pemphigus

agammaglobulinaemia tyrosine kinase
OrphanHuman

Overview

On 12 December 2017, orphan designation (EU/3/17/1951) was granted by the European Commission to Clinical Network Services (UK) Ltd, United Kingdom, for agammaglobulinaemia tyrosine kinase (also known as PRN1008) for the treatment of pemphigus.

The sponsorship was transferred to Clinical Network Services (NL) B.V., The Netherlands, in January 2019.

In August 2020, Clinical Network Services (NL) B.V.changed name to Scendea (NL) B.V.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2021 on request of the Sponsor.

Pemphigus is a group of autoimmune diseases characterised by widespread blistering and erosion of the skin and mucous membranes (the linings of internal organs). 'Autoimmune' means that the disease is caused by the immune system (the body's natural defences) attacking the body's own cells.

In pemphigus, the immune system produces antibodies that attack desmoglein, a protein which acts as a 'glue' attaching the cells of the epidermis (the outer layer of the skin). As a result skin cells separate from each other, causing blisters that turn into sores. In some cases these blisters can cover large areas of the skin.

Pemphigus is a long-term debilitating and life-threatening disease because of the long-term blistering which can lead to dehydration and infection.

At the time of designation, pemphigus affected approximately 1.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 72,000 people1, 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).

1Disclaimer: 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, prednisolone (a corticosteroid) and azathioprine (a medicine that reduces the activity of the immune system) were authorised in the EU to treat pemphigus.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with pemphigus because early studies show that it may reduce the need for corticosteroids in some patients. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine reduces the activity of an enzyme known as Bruton tyrosine kinase (BTK). This enzyme activates certain immune cells including those involved in pemphigus. By reducing BTK's activity, this medicine is expected to reduce the autoimmune response in patients with pemphigus, thus improving the symptoms of the disease.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with pemphigus were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for pemphigus. Orphan designation of the medicine had been granted in the United Stated for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 31 October 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.

EU/3/17/1951: Public summary of opinion on orphan designation: Agammaglobulinaemia tyrosine kinase for the treatment of pemphigus

Reference Number: EMA/COMP/737372/2017

English (EN) (181.3 KB - PDF)

First published: Last updated:
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Key facts

Active substance
agammaglobulinaemia tyrosine kinase
Intended use
Treatment of pemphigus
Orphan designation status
Withdrawn
EU designation number
EU/3/17/1951
Date of designation
Sponsor

Scendea (NL) B.V.

 

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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