EU/3/10/766 - orphan designation for treatment of ovarian cancer
Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2017 on request of the sponsor.
On 4 August 2010, orphan designation (EU/3/10/766) was granted by the European Commission to Æterna Zentaris GmbH, Germany, for Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt for the treatment of ovarian cancer.
Ovarian cancer is cancer of the ovaries (two organs in the female reproductive system that produce eggs). Most ovarian cancers occur in women over the age of 50 years. Due to the absence of symptoms in the early stages of the disease, the majority of patients are diagnosed when the cancer has spread locally or to other parts of the body.
Ovarian cancer is a life-threatening disease that is associated with poor long-term survival.
At the time of designation, ovarian cancer affected approximately 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 152,000 people, and is below the threshold 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 506,500,000 (Eurostat 2010).
At the time of designation, several medicines were authorised in the EU for the treatment of ovarian cancer. The choice of treatment depended mainly on how advanced the disease was. Treatments included surgery and chemotherapy (medicines to treat cancer).
The sponsor has provided sufficient information to show that Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2 might be of significant benefit for patients with ovarian cancer because it works in a different way to existing treatments, and early studies indicate that it might improve the treatment of patients with this condition, particularly patients who do not respond to existing treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2 is made of doxorubicin, a cytotoxic (cell-killing) agent, chemically attached to a modified form of a natural hormone, called luteinising hormone releasing hormone (LHRH, also known as gonadotropin releasing hormone, GnRH). The medicine is expected to work by attaching to the LHRH receptor, which is present on the surface of most ovarian cancer cells. Once attached, the doxorubicin is expected to kill the cancer cells, slowing down the growth and spread of the cancer.
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 Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2 in patients with ovarian cancer were ongoing.
At the time of submission, this medicine was not authorised anywhere in the EU for ovarian cancer. Orphan designation of the medicine had been granted in the United States of America for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 May 2010 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
- Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt
- Intended use
- Treatment of ovarian cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/10/766
- Date of designation
- Sponsor
Æterna Zentaris GmbH
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: