EU/3/16/1799 - orphan designation for treatment of tenosynovial giant cell tumour, localised and diffuse type
cabiralizumab
OrphanHuman
Overview
On 12 December 2016, orphan designation (EU/3/16/1799) was granted by the European Commission to Albany Regulatory Consulting Ltd, United Kingdom, for cabiralizumab for the treatment of tenosynovial giant cell tumour, localised and diffuse type.
The sponsorship was transferred to TMC Pharma Services Ltd, United Kingdom, in November 2017.
The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland in November 2019.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2020 on request of the Sponsor.
Tenosynovial giant cell tumour is a condition where the tissue surrounding the joints and tendons, called the synovial lining or synovium, expands abnormally forming outgrowths of the joint. It is known as 'localised' if only one site of the body is affected, or 'diffuse' when several sites are affected. It usually affects the hand joints of young adults and is characterised by pain, swelling and stiffness of the joint.
Tenosynovial giant cell tumour is a long-term debilitating disease because it causes the destruction of joints.
At the time of designation, tenosynovial giant cell tumour, localised and diffuse type affected less than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 154,000 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 513,700,000 (Eurostat 2016).
At the time of designation, no satisfactory methods were authorised in the EU for the treatment of tenosynovial giant cell tumour and treatment consisted of surgery and medicines to reduce inflammation.
Patients with tenosynovial giant cell tumours produce too much of a protein called colony-stimulating factor 1 (CSF1). This causes a build-up of immune cells called macrophages in joints, leading to the outgrowths.
Cabiralizumab attaches to and blocks the receptor to which CSF1 usually attaches, called CSF1R. This prevents CSF1 from working and is therefore expected to prevent outgrowths in the joints and thus delay the onset of symptoms of the disease.
The effects of cabiralizumab have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with cabiralizumab in patients with tenosynovial giant cell tumour, localised and diffuse type were ongoing.
At the time of submission, cabiralizumab was not authorised anywhere in the EU for tenosynovial giant cell tumour, localised and diffuse. Orphan designation of the medicine had been granted in the United States for pigmented villonodular synovitis and for tenosynovial giant cell tumor.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 November 2016 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
- cabiralizumab
- Intended use
- Treatment of tenosynovial giant cell tumour, localised and diffuse type
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/16/1799
- Date of designation
- Sponsor
TMC Pharma (EU) Limited
Durands Court 45
7a Parnell Street
Waterford
Co. Waterford
X91 P381
Ireland
Tel. +353 1 901 5595
E-mail: info@tmcpharma.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:
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: