Refusal of orphan designation for the treatment of epidermolysis bullosa



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
Intended use
Treatment of epidermolysis bullosa
Orphan designation status
EU designation number
Date of refusal of designation

Therapicon S.r.l.
Via Dei Grimani 10
20144 Milan
Tel. +39 02 433042

Review of designation

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

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

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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