EU/3/17/1931 - orphan designation for treatment in solid organ transplantation

siplizumab

Overview

On 16 October 2017, orphan designation (EU/3/17/1931) was granted by the European Commission to ITB-MED AB, Sweden, for siplizumab for the treatment in solid organ transplantation.

The sponsor’s address was updated in April 2019.

What is solid organ transplantation?

Solid organ transplantation is a surgical procedure in which a diseased organ, such as the heart, lungs, liver or kidney, is replaced with an organ from a donor.

Transplantation is a very complex procedure. During transplantation, the organ to be transplanted can become damaged because of the interruption and restoration of blood supply to the organ. In addition, graft rejection can occur after transplantation, when the recipient's body rejects the transplanted organ. Graft rejection is caused by the patient's immune system (the body's natural defences) recognising the transplanted graft as 'foreign' and attacking it.

These complications can be debilitating and life-threatening because they may result in the transplanted organ not working properly.

What is the estimated number of patients receiving solid organ transplants?

At the time of designation, approximately 1 in 10,000 people in the European Union (EU) were undergoing solid organ transplantation every year. This was equivalent to a total of around 52,000 people per year¹, and is below the ceiling for orphan designation. 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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¹Disclaimer: 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, several medicines were authorised in the EU to prevent or treat graft rejection in solid organ transplantation. These include antibodies such as antilymphocyte immunoglobulin and thymoglobulin and other medicines that suppress immune processes such as azathioprine, ciclosporin, mycophenolate mofetil, tacrolimus, and corticosteroids such as prednisolone or methylprednisolone.

The sponsor has provided sufficient information to show that siplizumab might be of significant benefit for patients undergoing solid organ transplantation. Early studies indicate that the medicine could help the recipient's body accept the transplant in the long term, allowing the recipient to stop taking medicines that suppress the immune system.

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?

Siplizumab is a monoclonal antibody (a type of protein) designed to recognise and attach to a protein called CD2 on the surface of T-lymphocytes, a type of white blood cell. When siplizumab attaches to CD2, it stops the white blood cells from recognising the transplanted organ as foreign, thereby reducing the immune system's attack on the transplanted organ. This is expected to decrease organ damage and lower the risk of the transplanted organ not working.

What is the stage of development of this medicine?

The effects of siplizumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with siplizumab in patients with solid organ transplants were ongoing.

At the time of submission, siplizumab was not authorised anywhere in the EU for use in patients with solid organ transplants or designated as an orphan medicinal product elsewhere for this use.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 September 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/3/17/1931: Public summary of opinion on orphan designation: Siplizumab for the treatment in solid organ transplantation

Reference Number: EMA/632175/2017

Key facts

• Active substance:
  siplizumab
• Intended use:
  Treatment in solid organ transplantation
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1931
• Date of designation:
  16/10/2017
• Sponsor:

  ITB-MED AB; Sonja Kovalevskys Gata 4; 113 66 Stockholm; Sweden; 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.