Refusal of orphan designation for treatment of epidermolysis bullosa

Diacerein
OrphanHuman

Overview

On 8 November 2018, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on the orphan designation application for diacerein for the treatment of epidermolysis bullosa. A negative decision was issued by the European Commission on 22 March 2019.

The sponsor applied for orphan designation on the basis of the seriousness and the rarity of the condition.

The negative opinion was based on the following reason:

  • The sponsor failed to demonstrate that the medicine could successfully treat patients with epidermolysis bullosa. The sponsor presented evidence from the scientific literature showing that a different formulation of diacerein was effective at treating blisters in patients with the condition; however, it did not demonstrate that the proposed formulation would be equally effective.

Requests for designation as an orphan medicinal product are made for investigational products. Absence of orphan designation does not preclude the development of this product, including its use in clinical trials. A marketing authorisation can still be obtained if quality, safety and efficacy are demonstrated.

Key facts

Active substance
Diacerein
Intended use
Treatment of epidermolysis bullosa
Orphan designation status
Negative
EU designation number
-
Date of refusal of designation
Sponsor

Therapicon S.r.l.
Via Dei Grimani 10
20144 Milan
Italy
Tel. +39 02 433042
E-mail: therapic@therapicon.com

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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