EU/3/14/1311 - orphan designation for treatment of Schnitzler syndrome
Gevokizumab
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2018 on request of the Sponsor.
On 22 August 2014, orphan designation (EU/3/14/1311) was granted by the European Commission to Les Laboratoires Servier, France, for gevokizumab for the treatment of Schnitzler syndrome.
The sponsorship was transferred to XOMA UK Limited, United Kingdom, in February 2016.
Schnitzler syndrome is a disease characterised by long-term urticaria (hives), monoclonal gammopathy (abnormal amounts of certain proteins in the blood), recurrent fever, bone and joint pain, and swollen lymph nodes. The disease usually appears during adulthood. Its cause is unclear.
Schnitzler syndrome is a long-term debilitating and life-threatening disease as it may lead to complications, including severe anaemia (low red blood cell counts), amyloidosis (build-up of abnormal protein deposits) and lymphoproliferative disorders (where white blood cells are produced in excessive amounts).
At the time of designation, Schnitzler syndrome affected approximately 0.02 in 10,000 people in the European Union (EU). This was equivalent to a total of around 1,000 people*, 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).
*Disclaimer: 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 511,100,000 (Eurostat 2014).
At the time of designation, no satisfactory methods were authorised for the treatment of Schnitzler syndrome. Corticosteroids and anti-inflammatory medicines known as NSAIDs were used to treat the symptoms of the disease. Anakinra (a medicine that works by blocking the receptor for a protein in the body called interleukin-1 beta) was also used.
Gevokizumab is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to the protein interleukin-1 beta. Interleukin-1 beta is involved in the process of inflammation and is thought to play a role in Schnitzler syndrome. By attaching to interleukin-1 beta, this medicine is expected to block its activity, helping to relieve the symptoms of the disease.
The effects of gevokizumab have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with gevokizumab in patients with Schnitzler syndrome were ongoing.
At the time of submission, gevokizumab was not authorised anywhere in the EU for Schnitzler syndrome or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 July 2014 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.
Key facts
- Active substance
- Gevokizumab
- Intended use
- Treatment of Schnitzler syndrome
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/14/1311
- Date of designation
- Sponsor
XOMA UK Limited
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: