EU/3/13/1169 - orphan designation for treatment of osteosarcoma
cisplatin
OrphanHuman
Overview
On 5 August 2013, orphan designation (EU/3/13/1169) was granted by the European Commission to Richardson Associates Regulatory Affairs Ltd, United Kingdom, for lipid-complexed cisplatin for the treatment of osteosarcoma.
The sponsorship was transferred to Richardson Associates Regulatory Affairs Ltd, Ireland, in December 2018.
Osteosarcoma is a type of cancer that affects the bone-forming cells. It usually affects the growing bone and occurs mainly in teenagers and young adults. Although it can develop in any bone in the body, it most often starts in the bones around the knee joint in the area of bone with the fastest growth. The commonest symptom is pain, which may be worse at night. Symptoms also depend on where the cancer has spread to. The most common site of spread for this cancer is the lungs.
Osteosarcoma is a long-term debilitating and life-threatening disease that may require patients to undergo limb amputations and is associated with poor long-term survival.
At the time of designation, osteosarcoma affected less than 2.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 127,000 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, the main treatments for osteosarcoma were surgery and chemotherapy (medicines to treat cancer). The goal of surgery was to remove cancerous bone while preserving as much skeletal function as possible. Chemotherapy was normally used to shrink the tumour before surgery and also after surgery to stop any remaining cancer cells from spreading. A number of chemotherapy medicines, including cisplatin, were authorised in the EU for this condition.
The sponsor has provided sufficient information to show that lipid-complexed cisplatin might be of significant benefit to patients with osteosarcoma because early studies show that it might be effective in treating osteosarcoma that has spread to the lungs and has come back after treatment with standard medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Lipid-complexed cisplatin is a new formulation of cisplatin, an anticancer medicine that has been used for osteosarcoma for several years. Cisplatin belongs to a class of medicines called 'alkylating agents' and works by attaching to the DNA of cells while they are reproducing, which stops cell division. As a result, cancer cells cannot divide and eventually die, slowing down the growth of tumours.
In this medicine, cisplatin is attached to lipid (fat) molecules so that the medicine can be inhaled into the lungs where cisplatin is then released gradually over time to act against cancer cells that that have spread to the lungs. The fat molecules attached to cisplatin are expected to keep the cisplatin in the patient's lungs for longer than 'free' cisplatin and thereby allow it to act over a longer period of time.
The effects of lipid-complexed cisplatin 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 osteosarcoma were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for osteosarcoma. Orphan designation had been granted in the United States for osteosarcoma that had spread to the lungs.
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.
Key facts
- Active substance
- cisplatin
- Intended use
- Treatment of osteosarcoma
- Orphan designation status
- Positive
- EU designation number
- EU/3/13/1169
- Date of designation
- Sponsor
Richardson Associates Regulatory Affairs Ltd
22 Northumberland Road
Ballsbridge
Dublin 4 D04 ED73
Ireland
Tel. + +353 6641177
E-mail: info@richardsonassociatesra.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
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: