EU/3/19/2139 - orphan designation for treatment of non-traumatic osteonecrosis
autologous adult live cultured osteoblasts
OrphanHuman
Overview
On 26 February 2019, orphan designation (EU/3/19/2139) was granted by the European Commission to Clinical Network Services (UK) Limited, United Kingdom, for autologous adult live cultured osteoblasts for the treatment of non-traumatic osteonecrosis.
The sponsorship was transferred to Clinical Network Services (NL) B.V., the Netherlands, in March 2019.
In August 2020, Clinical Network Services (NL) B.V.changed name to Scendea (NL) B.V.
Non-traumatic osteonecrosis is a disease characterised by loss and damage of bone tissue that is not caused by physical trauma but by the osteoblasts (bone-forming cells) not working properly. The disease mainly affects the hips, but can also affect other bones and joints such as the knees, jaws, shoulders and ankles. Symptoms include pain which becomes worse as the disease progresses and causes limitation in movements.
Non-traumatic osteonecrosis is a long-term debilitating condition, as patients become progressively unable to walk.
At the time of designation, non-traumatic osteonecrosis affected approximately 3.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 176,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 518,400,000 (Eurostat 2019).
At the time of designation, no satisfactory methods were authorised in the EU for the treatment of non-traumatic osteonecrosis. Medicines that widen blood vessels can reduce the pain in the early stage of the disease. Some patients were treated with surgery.
The medicine is made of cells that are extracted from the patient’s bone marrow and grown in a laboratory to form osteoblasts. Once implanted back into the patient’s bone, the cells are expected to replace the damaged bone tissue thereby relieving the symptoms of the disease.
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 non-traumatic osteonecrosis had finished.
At the time of submission, the medicine was authorised in India for the treatment of avascular necrosis (death of bone tissue due to interruption of blood supply) of the hip, under the name Ossgrow.
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 24 January 2019 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.
Key facts
- Active substance
- autologous adult live cultured osteoblasts
- Intended use
- Treatment of non-traumatic osteonecrosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/19/2139
- Date of designation
- Sponsor
Scendea (NL) B.V.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: