• Email
  • Help

Orphan designation

On 9 December 2011, orphan designation (EU/3/11/933) was granted by the European Commission to Merrimack Pharmaceuticals UK Limited, United Kingdom, for nanoliposomal irinotecan for the treatment of pancreatic cancer.

The sponsorship was transferred to Baxter Innovations GmbH, Austria, in December 2014.

In May 2015, Baxter Innovations GmbH changed name to Baxalta Innovations GmbH.

Nanoliposomal irinotecan has been authorised in the EU as Onivyde since 14 October 2016.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

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 1.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 71,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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.
At the time of designation, this represented a population of 507,700,000 (Eurostat 2011).

What methods of treatment 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 nanoliposomal irinotecan might be of significant benefit for patients with pancreatic cancer because it is a new formulation of irinotecan (an anticancer medicine authorised for the treatment of colorectal cancer), which is expected to improve the treatment of patients with this condition. 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?

Irinotecan is an anticancer medicine that belongs to the group ‘topoisomerase inhibitors’. It blocks an enzyme called topoisomerase I, which is involved in the division of cell DNA. When the enzyme is blocked, the DNA strands break. This prevents the cancer cells from dividing and they eventually die.

Free irinotecan is already authorised for the treatment of colorectal cancer. In this medicine, irinotecan is contained within tiny fat particles called ‘nanoliposomes’. The nanoliposomes are expected to accumulate within the tumour and release the medicine slowly over time, thereby decreasing the rate at which the irinotecan is removed from the body and allowing it to act for longer.

What is the stage of development of this medicine?

The effects of nanoliposomal irinotecan have been evaluated in experimental models.

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

At the time of submission, nanoliposomal irinotecan was not authorised anywhere in the EU for the treatment of pancreatic cancer. Orphan designation of nanoliposomal irinotecan had been granted in United States of America for pancreatic cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 October 2011 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>Nanoliposomal irinotecan</p>
Active substanceNanoliposomal irinotecan
Medicine NameOnivyde
Disease/conditionTreatment of pancreatic cancer
Date of decision09/12/2011
Orphan decision numberEU/3/11/933

Review of designation

During its meeting of 6-8 September 2016, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/11/933 for Onivyde (irinotecan1), as an orphan medicinal product for the treatment of pancreatic cancer. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with pancreatic cancer. The COMP recommended that the orphan designation of the medicine be maintained2.

1Previously known as liposomal irinotecan.

2The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.

Life-threatening or long-term debilitating nature of the condition

The Committee for Medicinal Products for Human Use (CHMP) recommended the authorisation of Onivyde for:

‘treatment of metastatic adenocarcinoma of the pancreas, in combination with 5‑fluorouracil (5‑FU) and leucovorin (LV), in adult patients who have progressed following gemcitabine based therapy’.

Metastatic adenocarcinoma of the pancreas is a form of pancreatic cancer. The CHMP indication therefore falls within the scope of the product’s designated orphan indication, which is: ‘pancreatic cancer’.

The COMP concluded that there had been no change in the seriousness of the condition since the orphan designation in 2011. Pancreatic cancer remains a life-threatening condition that is associated with shortened life expectancy.

Prevalence of the condition

The sponsor provided updated information on the prevalence of pancreatic cancer based on data from the European Cancer Observatory (IARC), the Globocan 2012 database, and the published literature.

On the basis of the information provided by the sponsor and the knowledge of the COMP, the COMP concluded that the prevalence of pancreatic cancer remains below the ceiling for orphan designation, which is 5 people in 10,000. At the time of the review of the orphan designation, the prevalence was estimated to be approximately 1.6 people in 10,000. This is equivalent to a total of around 82,000 people in the EU.

Existence of other methods of treatment

The COMP noted that, at the time of the review of the orphan designation, the main treatments for pancreatic cancer included the medicines mitomycin-c, 5‑fluorouracil, gemcitabine, erlotinib and protein-bound nanoparticle paclitaxel. These medicines were given to patients who had not been treated before (first-line therapy).

Significant benefit of Onivyde

The COMP concluded that the claim of a significant benefit of Onivyde in pancreatic cancer is justified on the basis of the results of a clinical study in patients with metastatic adenocarcinoma of the pancreas who had been previously treated with gemcitabine-based therapy. No medicines are currently authorised for these patients. The study showed that Onivyde used together with 5-fluorouracil and leucovorin prolonged patients’ lives.

Therefore, although other methods for the treatment of this condition have been authorised in the EU, the COMP concluded that Onivyde is of significant benefit to patients affected by pancreatic cancer.


Based on the data submitted and the scientific discussion within the COMP, the COMP considered that Onivyde still meets the criteria for designation as an orphan medicinal product and that Onivyde should remain in the Community Register of Orphan Medicinal Products.

Related information

Sponsor’s contact details

Baxalta Innovations GmbH
Industriestrasse 67
1221 Vienna
Tel. +43 1 20 100 247 2542
Fax +43 1 20 100 247 5990

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.