EU/3/13/1176: Orphan designation for the treatment of non-traumatic osteonecrosis

Non-traumatic osteonecrosis is a disease characterised by loss and damage of bone tissue that is not caused by physical trauma. In this disease, the bone-forming (osteoblastic) cells do not work properly, leading to the loss and damage of bone tissue. The disease mainly affects the hips, but can also affect other bones and joints such as the knees, shoulders and ankles. Symptoms include pain which becomes worse as the disease progresses and causes limitation in movement of the affected joints.

Non-traumatic osteonecrosis is a long-term debilitating condition, as patients become progressively unable to walk due to pain.

At the time of designation, non-traumatic osteonecrosis affected approximately 2.9 in 10,000 people in the European Union (EU). This was equivalent to a total of around 148,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of non-traumatic osteonecrosis. Some patients were treated with surgery.

This medicine is an advanced medicinal product that belongs to the group called 'tissue engineered products'. These are medicines that contain cells or tissues that have been 'engineered' (modified) so they can be used to repair, regenerate or replace tissue.

The medicine is made of osteoblastic-like cells extracted from the bone marrow of a donor and grown in a laboratory. Once implanted into the patient's bone, the cells are expected to replace the damaged bone tissue thereby relieving the symptoms of the disease.

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicinal product in experimental models was ongoing.

At the time of submission, no clinical trials with the medicinal product in patients with non-traumatic osteonecrosis had been started.

At the time of submission, the medicinal product was not authorised anywhere in the EU for non-traumatic osteonecrosis 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 11 July 2013 recommending the granting of this designation.

• 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.