EU/3/15/1488 - orphan designation for treatment of ovarian cancer

{2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide

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Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2017 on request of the sponsor.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 21 May 2015, orphan designation (EU/3/15/1488) was granted by the European Commission to Right Track Regulatory Limited, United Kingdom, for {2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide for the treatment of ovarian cancer.

The sponsorship was transferred to Celgene Europe Limited in April 2017.

What is ovarian cancer?

Ovarian cancer is cancer of the ovaries, the two organs in the female reproductive system that produce egg cells. 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 to other parts of the body.

Ovarian cancer is a long-term debilitating and 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 less than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 154,000 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).

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*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 512,900,000 (Eurostat 2015).

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 this medicine might be of significant benefit for patients with ovarian cancer because it works in a different way to existing treatment and results from experimental studies suggest that use with existing treatment (i.e. pegylated liposomal doxorubicin) could increase its effectiveness. 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?

The medicine acts on certain receptors (known as Toll-like receptors) found on cells of the immune system (the body's natural defences). By its action on these receptors, the medicine is expected to stimulate the immune cells, increasing the ability of the immune system to recognise and attack cancer cells. When given in combination with other cancer treatments, the medicine is expected to help kill the cancer cells.

What is the stage of development of this medicine?

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with ovarian cancer were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for ovarian cancer. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 April 2015 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.

EU/3/15/1488: Public summary of opinion on orphan designation: {2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide for the treatment of ovarian cancer

Adopted Reference Number: EMA/COMP/269491/2015

Key facts

• Active substance:
  {2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide
• Intended use:
  Treatment of ovarian cancer
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/15/1488
• Date of designation:
  21/05/2015
• Sponsor:

  Celgene Europe Limited; 1 Longwalk Road; Stockley Park; Uxbridge; Middlesex UB11 1DB; United Kingdom; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.