EU/3/07/490 - orphan designation for treatment of non-traumatic osteonecrosis

human autologous bone-forming cells derived from bone marrow stem cells

Overview

On 29 October 2007, orphan designation (EU/3/07/490) was granted by the European Commission to Bone Therapeutics SA, Belgium, for human autologous bone-forming cells derived from bone marrow stem cells for treatment of non-traumatic osteonecrosis.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2019 on request of the Sponsor.

What is non-traumatic osteonecrosis?

Osteonecrosis is a slow progressive disorder characterized by bone destruction most commonly in the head of the femur (the long bone in the thigh), although other bones can be involved. Non-traumatic osteonecrosis is generally associated with one or more risk factors, and in over two thirds of patients it is related to glucocorticoid therapy and/or alcohol abuse. As the disease progresses, most patients experience pain in the groin, in the thigh or even in the knee, and eventually develop osteoarthritis of the hip. The condition is chronically debilitating, as patients become progressively unable to walk due to pain and arthritis.

What are the methods of treatment available?

There are no authorised products for the condition in the Community. Surgical management is often required.

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

According to the information provided by the sponsor, non-traumatic osteonecrosis was considered to affect about 100,000 persons in the European Union.

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

Bone is a living tissue, and it is constantly destroyed (resorbed) and rebuilt (formed) in a process called bone remodelling. Bone remodelling is done by two types of cells: the osteoclasts for resorption and the osteoblasts for formation. Human bone marrow stem cells are collected from the same individual (autologous), and transformed into human autologous bone forming cells, under not fully understood mechanisms. It is hypothesized that human autologous bone forming cells will restore the bone of the patients.

What is the stage of development of this medicinal product?

The evaluation of the effects of human autologous bone-forming cells derived from bone marrow stem cells in experimental models was ongoing.

At the time of submission of the application for orphan designation, clinical trials in patients with nontraumatic osteonecrosis were ongoing.

Human autologous bone-forming cells derived from bone marrow stem cells were not authorised anywhere in the world for treatment of non-traumatic osteonecrosis or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 12 September 2007 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:

• the seriousness of the condition,
• the existence or not of alternative methods of diagnosis, prevention or treatment and
• either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

EU/3/07/490: Public summary of positive opinion for orphan designation of human autologous bone-forming cells derived from bone marrow stem cells for treatment of non-traumatic osteonecrosis

Adopted Reference Number: EMEA/COMP/440983/2007

Key facts

• Active substance:
  human autologous bone-forming cells derived from bone marrow stem cells
• Intended use:
  Treatment of non-traumatic osteonecrosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/07/490
• Date of designation:
  29/10/2007
• Sponsor:

  Bone Therapeutics SA; Rue Auguste Piccard 37; 6041 Gosselies; Belgium; Tel. [phone]; Fax [phone]; E-mail: [e-mail]