EU/3/09/695

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Orphan designation

On 30 November 2009, orphan designation (EU/3/09/695) was granted by the European Commission to Merck Sharp & Dohme Limited, United Kingdom, for 8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride 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 leads to 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 151,000 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 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 504,800,000 (Eurostat 2009).

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 is. Treatments included surgery and chemotherapy (medicines to treat cancer).
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with ovarian cancer because it works in a different way to existing medicines and because early studies in experimental models indicate that it might improve the treatment of patients with this condition. These assumptions 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?

8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride is expected to work by blocking types of enzymes called AKT. These enzymes are protein kinases which are involved in cell survival and growth. In ovarian cancer, AKT enzymes are often activated, enabling cancer cells to survive and multiply. By blocking AKT, this medicine is expected to control cell survival and slow down the growth of cancer cells.

What is the stage of development of this medicine?

The effects of 8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the designated product including patients with ovarian cancer were ongoing.
At the time of submission, 8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride was not authorised anywhere in the EU for ovarian cancer. Orphan designation of this medicine had been granted in the United States of America for ovarian cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 October 2009 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 Community) 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>8-[4-(1-Aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride</p>
Active substance8-[4-(1-Aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision29/11/2009
OutcomePositive
Orphan decision numberEU/3/09/695

Review of designation

Sponsor’s contact details:

Merck Sharp & Dohme Limited
Hertford Road
Hoddesdon
Hertfordshire, EN11 9BU
United Kingdom
Telephone: +32 2 776 60 45
Telefax: +32 2 776 62 88
E-mail: axel_breitstadt@merck.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