Refusal of orphan designation for the treatment of status epilepticus

Midazolam Hydrochloride

Table of contents


On 30 July 2003, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on orphan designation of midazolam hydrochloride (for oromucosal use) for the treatment of seizures which continue for at least five minutes. A negative decision was granted by the European Commission on 1 March 2004.

The sponsor applied for orphan designation on the basis of severity and rarity of the condition, as well as an assumption of potential benefit despite currently available methods of treatment.

The negative opinion is based on the following element:

The sponsor has not established that seizures which continue for at least five minutes affect not more than 5 in 10,000 persons in the Community at the time the application was made.*

Requests for designation as orphan medicinal products are made for investigational products. Absence of orphan designation does not preclude the development of this product, in particular through clinical trials, and subsequently the possibility of obtaining a marketing authorisation if quality, safety and efficacy are demonstrated.

*Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 385,000,000 (Eurostat 2002) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.

Key facts

Active substance
Midazolam Hydrochloride
Intended use
Treatment of status epilepticus
Orphan designation status
EU designation number
Date of refusal of designation
Special Products Ltd.
Orion House, 49 High Street
Surrey KT15 1TU
United Kingdom
Telephone: +44 19 32 82 06 66
Telefax: +44 19 32 85 04 44

Documents related to this orphan designation evaluation

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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