EU/3/01/048

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Orphan designation

On 9 July 2001, orphan designation (EU/3/01/048) was granted by the European Commission to Elan Pharma International Ltd, Ireland, for ziconotide (intraspinal use) for the treatment of chronic pain requiring intraspinal analgesia. The sponsorship was transferred to Eisai Limited, United Kingdom, in September 2006.

What is chronic pain requiring intraspinal analgesia?

Chronic pain is often defined as pain that persists for more than three months. Chronic pain has no time limit. The most common conditions associated with chronic pain include low-back pain, headache, recurrent pain in the face, pain associated with cancer, and arthritic pain. Drug therapy, mainly using products called analgesics, is used to ease chronic pain. Analgesics may be administered directly to the spinal cord, inside the spine (intraspinal administration), when the pain cannot be controlled by other routes of administration, such as with tablets, skin patches, or injections. Chronic pain requiring intraspinal analgesia is chronically debilitating.

What are the methods of treatment available?

At the time of submission of application for orphan drug designation, treatment of chronic pain requiring intraspinal analgesia consisted of authorised products (morphine and other opioids, bupivacaine) in the European Union.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that ziconotide (intraspinal use) might be of potential significant benefit for the treatment of chronic pain requiring intraspinal analgesia. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

What is the estimated number of patients affected by the condition*?

According to the information provided by the sponsor, chronic pain requiring intraspinal analgesia was considered to affect about 58,000 persons in the European Union.


*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 377,000,000 (Eurostat 2001) 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.

How is this medicinal product expected to act?

Ziconotide is a man-made equivalent of a molecule composed of a 25-amino acid chain, which is found in the venom of a fish-eating marine snail, Conus magus. Ziconotide blocks the action of specific molecules found in nerve tissue, called N-type voltage-sensitive calcium channels. These molecules play an important role in chronic pain. Ziconotide is expected to decrease pain by blocking the function of these molecules.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, the effects of ziconotide (intraspinal use) had been evaluated in experimental models, and clinical trials in patients with chronic pain requiring intraspinal analgesia were ongoing.

Ziconotide (intraspinal use) was not marketed anywhere in the world for the treatment of chronic pain requiring intraspinal analgesia, nor designated as an orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 23 May 2001 a positive opinion recommending the grant of the above-mentioned designation.

Update: Ziconotide (intraspinal use) (Prialt) is authorised in the European Union as of 21 February 2005 for the treatment of severe, chronic pain in patients who require intrathecal (IT) analgesia.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition,
  • the existence or not of alternative methods of diagnosis, prevention or treatment and
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

Key facts

Product details for <p>Ziconotide (intraspinal use)</p>
Active substanceZiconotide (intraspinal use)
Medicine NamePrialt
Disease/conditionTreatment of chronic pain requiring intraspinal analgesia
Date of decision09/07/2001
OutcomePositive
Orphan decision numberEU/3/01/048

Review of designation

Related information

Sponsor’s contact details

Eisai Ltd
European Knowledge Centre
Mosquito Way
Hatfield
Herts
AL10 9SN
United Kingdom
Tel. +44 (0)8456 761400
Fax +44 (0)8456 761401

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.