EU/3/15/1464 - orphan designation for treatment of systemic sclerosis
autologous adipose tissue-derived stromal vascular fraction cells
OrphanHuman
Overview
On 19 March 2015, orphan designation (EU/3/15/1464) was granted by the European Commission to Assistance Publique Hôpitaux de Marseille, France, for autologous adipose tissue-derived stromal vascular fraction cells for the treatment of systemic sclerosis.
Systemic sclerosis is a complex disease in which the immune system (the body's natural defences) is overactive, causing inflammation and excess production of various proteins, particularly collagen. The reason why the immune system is overactive is not known. Collagen is an important component of connective tissue (the tissue that supports the skin and internal organs).
The overproduction of collagen leads to the abnormal growth of connective tissue, causing the skin to become thick and hard (fibrosis). Initial symptoms include swollen fingers and hands, followed by thickened skin over the arms, legs, face and trunk. The disease can also damage the walls of the blood vessel of the internal organs, such as the heart, lungs and kidneys. This makes it more difficult for the blood to flow, causing tissue damage and circulation problems.
Systemic sclerosis is a long-lasting, debilitating disease and may be life threatening because of its possible effects on the gut, heart, lungs and kidneys.
At the time of designation, systemic sclerosis affected less than 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 180,000 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 512,900,000 (Eurostat 2015).
At the time of designation, there were no treatments for systemic sclerosis that could stop the build-up of collagen. Treatments authorised in the EU were aimed at relieving the symptoms of the disease and limiting the damage it causes. Several medicines were used to reduce inflammation and circulation problems. Bosentan was authorised in the EU specifically to treat patients with systemic sclerosis in whom poor blood circulation caused by the disease has led to the development of 'digital ulcers' (sores on the fingers and toes).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with systemic sclerosis because early studies showed that it may be beneficial to patients who did not respond to other treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine is prepared from the patient's own body fat (adipose tissue). The fat is broken down in the laboratory using digestive enzymes, and the resulting tissue is devoid of fat cells but contains other cells such as leukocytes and macrophages (types of white blood cells), and stem cells (cells that can develop into different types of cell). When these remaining cells are injected back into the patient's hands, it is expected that they will help promoting tissue repair in the damaged areas, helping to slow down or stop the progression of the disease in the treated area.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with systemic sclerosis were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for systemic sclerosis 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 12 February 2015 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
- autologous adipose tissue-derived stromal vascular fraction cells
- Intended use
- Treatment of systemic sclerosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/15/1464
- Date of designation
- Sponsor
Assistance Publique Hôpitaux de Marseille
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: