EU/3/15/1499 - orphan designation for treatment of thromboangiitis obliterans (Buerger's disease)
Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells
OrphanHuman
Overview
On 21 May 2015, orphan designation (EU/3/15/1499) was granted by the European Commission to Regulatory Resources Group Ltd, United Kingdom, for adult human bone-marrow derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells for treatment of thromboangiitis obliterans (Buerger's disease).
The sponsorship was transferred to Voisin Consulting S.A.R.L., France, in February 2019.
In October 2021, Voisin Consulting S.A.R.L. changed name to Voisin Consulting Life Sciences.
Thromboangiitis obliterans, also known as Buerger's disease, is a disease in which the blood vessels become inflamed. Because of the inflammation, clots form within the blood vessels, blocking them and causing pain. The small blood vessels in the limbs (especially in the hands, legs and feet) are most often affected, and the lack of blood flow can cause skin ulcers (sores) and gangrene (decay and death of tissue). Ultimately, the limb may need to be amputated. Although the causes of the inflammation in thromboangiitis obliterans are not known, the disease is known to be closely linked to smoking.
Thromboangiitis obliterans is a long-term debilitating condition because of the development of ulcers and gangrene, and the risk of amputation.
At the time of designation, thromboangiitis obliterans affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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, iloprost was authorised in some EU Member States for the treatment of advanced thromboangiitis obliterans. Other medicines were also used to control the symptoms of the disease such as pain, and to promote the healing of skin ulcers. Vascular surgery (surgery on the blood vessels) was used in a small number of cases.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with thromboangiitis obliterans because early studies show that the product reduces pain during rest and heals ulcers in patients with the condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is made of cells, called mesenchymal stromal cells, extracted from the bone marrow of different adult donors, which are pooled together and grown in the laboratory. Mesenchymal stromal cells can produce several proteins important to regulate the immune system, control inflammation and the growth of cells. Although the mechanism by which the mesenchymal stromal cells in this medicine work is not fully known, the cells are expected to promote the growth of new blood vessels and tissue repair. This is expected to improve the blood supply in the limbs of patients with thromboangiitis obliterans, relieving the symptoms of the disease.
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 thromboangiitis obliterans were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for thromboangiitis obliterans 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 16 April 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
- Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells
- Intended use
- Treatment of thromboangiitis obliterans (Buerger's disease)
- Orphan designation status
- Positive
- EU designation number
- EU/3/15/1499
- Date of designation
- Sponsor
Voisin Consulting Life Sciences
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: