EU/3/18/2131 - orphan designation for treatment of immune thrombocytopenia

Rozanolixizumab

Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in May 2023 on request of the Sponsor.

On 11 January 2019, orphan designation (EU/3/18/2131) was granted by the European Commission to UCB Biopharma S.P.R.L., Belgium, for rozanolixizumab for the treatment of immune thrombocytopenia.

 

What is immune thrombocytopenia?

Immune thrombocytopenia is a condition in which the immune system (the body’s natural defences) attacks the platelets, components in the blood that help it to clot. As a result, blood levels of platelets are low (thrombocytopenia) resulting in spontaneous bleeding and bruising. Although not all patients experience bleeding, severe bleeding can occur in some patients.

Immune thrombocytopenia is a debilitating and life-threatening condition because of the risk of severe bleeding, especially in the brain.

What is the estimated number of patients?

At the time of designation, immune thrombocytopenia affected approximately 2.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 135,000 people¹, 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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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 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, authorised treatments included human normal immunoglobulin, Nplate (romiplostim), Revolade (eltrombopag) and certain corticosteroids. Some patients required surgery (splenectomy) to remove the spleen, an organ involved in the removal of platelets from the body.

The sponsor has provided sufficient information to show that rozanolixizumab might be of significant benefit for patients with immune thrombocytopenia. Early studies show that the medicine could lead to significant improvements in platelet levels in patients whose condition is difficult to treat. 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?

In patients with immune thrombocytopenia, components of the immune system called IgG antibodies are faulty and damage platelets. Rozanolixizumab is expected to remove the faulty IgG antibodies from the body by blocking a protein called FcRn on the surface of cells, which keeps these antibodies in the body for longer. By blocking FcRn, the medicine is expected to increase the levels of platelets in the blood and thus improve symptoms of the condition.

What is the stage of development of this medicine?

The effects of rozanolixizumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with rozanolixizumab in patients with immune thrombocytopenia were ongoing.

At the time of submission, rozanolixizumab was not authorised anywhere in the EU for immune thrombocytopenia. It was designated as an orphan medicinal product in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 December 2018 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/3/18/2131: Public summary of opinion on orphan designation: Rozanolixizumab for the treatment of immune thrombocytopenia

Reference Number: EMADOC-628903358-230

Key facts

• Active substance:
  Rozanolixizumab
• Intended use:
  Treatment of immune thrombocytopenia
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/18/2131
• Date of designation:
  11/01/2019
• Sponsor:

  UCB Biopharma; Allee De La Recherche 60; 1070 Anderlecht; Belgium; Tel. [phone]; Email: [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.