EU/3/17/1854 - orphan designation for treatment of thromboangiitis obliterans (Buerger's disease)

autologous adipose tissue-derived mesenchymal stem cells

Overview

On 20 March 2017, orphan designation (EU/3/17/1854) was granted by the European Commission to SPC GmbH, Germany, for autologous adipose tissue-derived mesenchymal stem cells (also known as VascoStem) for the treatment of thromboangiitis obliterans (Buerger's disease).

What is thromboangiitis obliterans (Buerger's disease)?

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 pain, the development of ulcers and gangrene, and the risk of amputation.

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

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 52,000 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 515,700,000 (Eurostat 2017).

What treatments are available?

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 results of early studies in patients who could not be given surgery suggest it increased the distance they could walk without pain. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine is composed of mesenchymal stem cells (cells that can develop into certain other types of cells such as muscle and bone cells) that are extracted from the patient's own fat tissue. After extraction they are grown in the laboratory to increase their number before being injected back into the patient.

Although the way they work in the condition is not fully known, these stem cells produce various substances that help the growth and repair of body tissues. It is expected that treatment with the medicine will therefore reduce damage to the blood vessels and so improve the symptoms of the condition.

What is the stage of development of this medicine?

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. Orphan designation had been granted in the United States for the condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 February 2017 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.

EU/3/17/1854: Public summary of opinion on orphan designation: Autologous adipose tissue-derived mesenchymal stem cells for the treatment of thromboangiitis obliterans (Buerger's disease)

Adopted Reference Number: EMA/138358/2017

Key facts

• Active substance:
  autologous adipose tissue-derived mesenchymal stem cells
• Intended use:
  Treatment of thromboangiitis obliterans (Buerger's disease)
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1854
• Date of designation:
  20/03/2017
• Sponsor:

  SPC GmbH; An der Kirche 4; 55130 Mainz; Germany; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.