EU/3/19/2181 - orphan designation for treatment of soft tissue sarcoma
7-ethyl-10-hydroxy-camptothecin
OrphanHuman
Overview
On 25 July 2019, orphan designation EU/3/19/2181 was granted by the European Commission to Cebiotex S.L., Spain, for 7-ethyl-10-hydroxy-camptothecin for the treatment of soft tissue sarcoma.
Soft tissue sarcoma is a cancer that affects the soft, supportive tissues of the body. It can occur in muscles, blood vessels, fat tissue or in other tissues that support, surround and protect organs. Patients with soft tissue sarcoma do not usually have symptoms in the early stages of the disease. First symptoms appear when the cancer grows large enough to cause swelling and pain.
Soft tissue sarcoma is a long-term debilitating and life-threatening disease, particularly when the cancer has spread to other parts of the body.
At the time of designation, soft tissue sarcoma affected approximately 3.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 187,000 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, the main treatments for soft tissue sarcoma were surgery and chemotherapy (medicines to treat cancer). Radiotherapy (treatment with radiation) was also used. Several medicines have been authorised in the EU for the treatment of soft tissue sarcoma.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with soft tissue sarcoma. Laboratory studies show that the medicine, given in combination with doxorubicin, a standard chemotherapy treatment for the condition, is more active against cancer cells than doxorubicin alone. In addition, the sponsor provided data from the literature showing that the medicine may be added to chemotherapy or radiotherapy. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
7-Ethyl-10-hydroxy-camptothecin is the active form of a well-known anti-cancer medicine called irinotecan. It belongs to the group ‘topoisomerase inhibitors’ and blocks an enzyme called topoisomerase I, which is involved in the division of DNA. When the enzyme is blocked, the DNA strands break. This prevents the cancer cells from dividing and they eventually die. The active substance is incorporated into a membrane that will be implanted into the patient’s tissue after they have received surgery. The membrane will allow the slow release of the cancer medicine into the remaining tissue where it will control and kill cancer cells.
The effects of 7-ethyl-10-hydroxy-camptothecin have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with 7-ethyl-10-hydroxy-camptothecin in patients with soft tissue sarcoma were ongoing.
At the time of submission, 7-ethyl-10-hydroxy-camptothecin was not authorised anywhere in the EU for the treatment of soft tissue sarcoma or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 20 June 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
- 7-ethyl-10-hydroxy-camptothecin
- Intended use
- Treatment of soft tissue sarcoma
- Orphan designation status
- Positive
- EU designation number
- EU/3/19/2181
- Date of designation
- Sponsor
Cebiotex S.L.
Calle De La Via Layetana 26, Tech Pier 07 Entresuelo
Barcelona
08003
Spain
E-mail: info@cebiotex.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| June 2023 | The sponsor’s address was updated in June 2023. |
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: