EU/3/16/1719 - orphan designation for treatment of malignant mesothelioma
cisplatin
Orphan
Human
Overview
On 29 August 2016, orphan designation (EU/3/16/1719) was granted by the European Commission to PlumeStars s.r.l., Italy, for cisplatin for the treatment of malignant mesothelioma.
Malignant mesothelioma is a cancer that affects the mesothelial cells (found on the inner linings of the organs), mainly in the pleura (the lining of the lungs) and in the peritoneum (the lining of the abdominal cavity). It is usually caused by exposure to asbestos. Mesothelioma of the pleura causes difficulty breathing and chest pain, and mesothelioma of the peritoneum causes ascites (a build-up of fluid in the abdomen) and abdominal pain.
Malignant mesothelioma is life-threatening because it may lead to bowel obstruction, heart or breathing problems and lung infections. Patients have very poor survival, only living for a year, on average, after diagnosis.
At the time of designation, malignant mesothelioma affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 51,000 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 513,700,000 (Eurostat 2016).
At the time of designation, the main treatments for malignant mesothelioma were surgery and chemotherapy (medicines to treat cancer) with or without radiotherapy (treatment with radiation). If the disease was too advanced for surgery, chemotherapy alone was used. One medicine, pemetrexed, was authorised throughout the EU for the treatment of malignant pleural mesothelioma. A second medicine, raltitrexed, was authorised for the treatment of malignant pleural mesothelioma in some European countries.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with malignant mesothelioma because early studies in experimental models showed that the medicine reduced any tumours left in the lungs after surgery. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Cisplatin is a cancer medicine that has been used to treat various types of cancer for several years. It belongs to a class of medicines called 'alkylating agents' and works by attaching to the DNA of cells, preventing them from multiplying. As a result, cancer cells eventually die, slowing down the growth of tumours.
This medicine consists of a thin and flexible film of material loaded with cisplatin, which is to be implanted in the affected lung during surgery to prevent tumour growth. The film releases cisplatin gradually over time to act against cancer cells that may not have been removed during surgery.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with malignant mesothelioma had been started.
At the time of submission, cisplatin was not authorised anywhere in the EU for malignant mesothelioma 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 13 July 2016 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 malignant mesothelioma
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1719
- Date of designation
- Sponsor
PlumeStars s.r.l.
Strada Inzani 1
43125 Parma
Italy
Tel. +39 0521 90 50 86
Fax +39 0521 90 50 06
E-mail: info@plumestars.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:
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: