EU/3/16/1643 - orphan designation for treatment of systemic sclerosis

autologous stromal vascular cell fraction from adipose tissue

Overview

On 28 April 2016, orphan designation (EU/3/16/1643) was granted by the European Commission to Cytori Ltd, United Kingdom, for autologous stromal vascular cell fraction from adipose tissue for the treatment of systemic sclerosis.

The sponsorship was transferred to Clinical Network Services (NL) B.V., Netherlanks, in March 2019.

In August 2020, Clinical Network Services (NL) B.V. changed name to Scendea (NL) B.V.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in August 2020 on request of the Sponsor.

What is systemic sclerosis?

Systemic sclerosis, also known as scleroderma, is a complex disease in which the immune system (the body's natural defences) is overactive, causing inflammation and excessive production of some 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).

Overproduction of collagen leads to abnormal growth of connective tissue, causing the skin to become thick and hard. 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 blood vessels of 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.

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

At the time of designation, systemic sclerosis affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 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).

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*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).

What treatments are available?

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 has been 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 this medicine might be of significant benefit in systemic sclerosis because preliminary studies suggest it can produce improvement in patients whose condition has not responded to existing treatments. 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 prepared from the patient's own body fat (adipose tissue). The fat is broken down in the laboratory using digestive enzymes to remove fat cells but leave behind other cells such as leukocytes and macrophages (types of white blood cells), and stem cells (cells that can develop into different types of cell). Injecting these cells back under the patient's skin is expected to improve blood supply and promote tissue repair in the damaged areas, helping to slow down or stop the progression of the disease in the treated area.

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 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 23 March 2016 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/3/16/1643: Public summary of opinion on orphan designation: Autologous stromal vascular cell fraction from adipose tissue for the treatment of systemic sclerosis

Adopted Reference Number: EMA/COMP/236625/2016

Key facts

• Active substance:
  autologous stromal vascular cell fraction from adipose tissue
• Intended use:
  Treatment of systemic sclerosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/16/1643
• Date of designation:
  28/04/2016
• Sponsor:

  Scendea (NL) B.V.

Review of designation

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