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Orphan designation

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in September 2017 on request of the sponsor.

On 17 July 2013, orphan designation (EU/3/13/1152) was granted by the European Commission to Merck KGaA, Germany, for (1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N’-bis(2-bromoethyl)diamidophosphate (also known as TH-302) for the treatment of pancreatic cancer.

The sponsorship was transferred to Chiltern International Limited, United Kingdom, in June 2016.

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).

*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 509,000,000 (Eurostat 2013).

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 have shown that it may improve the outcome of patients with this condition when used alone or in combination with 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?

Pancreatic cancer, like most solid tumours, has areas with poor blood supply and therefore low levels of oxygen. The low level of oxygen in these areas is known to make tumour cells more resistant to standard chemotherapy. This medicine is expected to be converted into an active, toxic form called bromo-isophosphoramide mustard under conditions of low oxygen, allowing it to attack the tumour cells in low oxygen areas. This medicine is intended to be given with standard chemotherapy medicines and this is expected to help kill the tumour cells in low oxygen areas as well as other areas of the tumours.

What is the stage of development of this medicine?

The effects of the medicinal product have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicinal product in patients with pancreatic cancer were ongoing.

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 13 June 2013 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>(1-Methyl-2-nitro-1H-imidazole-5-yl)methyl N,N&#8217;-bis(2-bromoethyl)diamidophosphate</p>
Active substance(1-Methyl-2-nitro-1H-imidazole-5-yl)methyl N,N’-bis(2-bromoethyl)diamidophosphate
Medicine Name
Disease/conditionTreatment of pancreatic cancer
Date of decision17/07/2013
Orphan decision numberEU/3/13/1152

Review of designation

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in September 2017 on request of the sponsor.

Sponsor’s contact details:

Chiltern International Limited
171 Bath Road
Berkshire SL1 4AA
Tel. +44 (0)1315 507 700

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.