EU/3/14/1412 - orphan designation for treatment of pancreatic cancer

Herpes simplex type 1 virus containing cellular B-myb gene as tumour-specific promoter

Overview

On 15 January 2015, orphan designation (EU/3/14/1412) was granted by the European Commission to Karcinolys S.A.S., France, for herpes simplex type 1 virus containing cellular B-myb gene as tumour-specific promoter for the treatment of pancreatic cancer.

What is pancreatic cancer?

Pancreatic cancer is cancer of the pancreas, a small organ that lies behind the stomach. The pancreas has two functions: to produce a juice that helps with the digestion of food, and to produce hormones such as insulin. Due to the absence of symptoms in the early stages of pancreatic cancer, the majority of patients are diagnosed when the cancer has spread locally or to other parts of the body.

Pancreatic cancer is a very severe and life-threatening disease that is associated with shortened life expectancy.

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

At the time of designation, pancreatic cancer affected approximately 2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 102,000 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 511,100,000 (Eurostat 2014).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for treating pancreatic cancer. The choice of treatment depended on several factors, including how far the disease had advanced. Treatments included surgery, radiotherapy (treatment with radiation) 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 pancreatic cancer because early studies in experimental models showed an anti-tumour effect, which was increased when the medicine was used together with the chemotherapy medicine gemcitabine. 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?

This medicine consists of an 'oncolytic' virus (a virus that kills cancer cells) that contains a part of a gene (called B-myb). This is expected to allow the virus to multiply and kill only cells that have high levels of the gene B-myb (such as pancreatic cancer cells), while sparing normal cells.

The virus in the medicine (a herpes virus) has been modified so that it does not cause disease in humans.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with pancreatic cancer had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for pancreatic cancer or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 December 2014 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/3/14/1412: Public summary of opinion on orphan designation: Herpes simplex type 1 virus containing cellular B-myb gene as tumour-specific promoter for the treatment of pancreatic cancer

Adopted Reference Number: EMA/COMP/797466/2014

Key facts

• Active substance:
  Herpes simplex type 1 virus containing cellular B-myb gene as tumour-specific promoter
• Intended use:
  Treatment of pancreatic cancer
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/14/1412
• Date of designation:
  15/01/2015
• Sponsor:

  Karcinolys S.A.S.; 16, rue de Pervenches; 92500 Rueil-Malmaison; France; Tel. [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.