EU/3/10/766

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Orphan designation

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.

What is 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.

What is the estimated number of patients affected by the condition?

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).

What treatments are available?

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.

How is this medicine expected to work?

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.

What is the stage of development of this medicine?

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.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • 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

Product details for <p>Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt</p>
Active substancePyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision04/08/2010
OutcomePositive
Orphan decision numberEU/3/10/766

Review of designation

Sponsor’s contact details:

Æterna Zentaris GmbH
Weismüllerstr. 50
60314 Frankfurt am Main
Germany
Telephone: +49 69 426 02 34 75
Telefax: +49 69 42 602 34 04
E-mail: info@aezsinc.com

Patient associations’ contact points:

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
France
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10
E-mail: ligue@ligue-cancer.net

Macmillan Cancer Support
3 Bath Place
Rivington Street
London EC2A 3JR
United Kingdom
Telephone: +44 20 7696 9003
Telefax: +44 20 7696 9002

European Cancer Patient Coalition
ECPC Office
Am Rothenanger 1b
85521 Riemerling
Germany
Telephone: +49 89 628 36 807
Telefax: +49 89 628 36 808
E-mail: info@ecpc-online.org