EU/3/14/1394

On 16 December 2014, orphan designation (EU/3/14/1394) was granted by the European Commission to Pharm. Research Associates (UK) Limited, United Kingdom, for pegylated recombinant human hyaluronidase PH20 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 fluid 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 1.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 92,000 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).

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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 the medicine might be of significant benefit for patients with pancreatic cancer because early results suggest that response rates and survival times may be increased in patients with pancreatic cancer that has spread to other parts of the body who are given this medicine in addition to conventional treatment. 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 contains a version of a natural enzyme, hyaluronidase, which breaks down a compound, hyaluronan. Hyaluronan is found in large amounts in many pancreatic cancers and helps the cancer to grow and to resist the effects of cancer medicines. By breaking down the excess hyaluronan, the medicine is expected to make the cancer easier to treat with other authorised therapies.

In this medicine, hyaluronidase has been ‘pegylated’ (combined with a chemical called polyethylene glycol). This decreases the rate at which the substance is removed from the body and allows the medicine to be given less often.

What is the stage of development of this medicine?

The effects of pegylated recombinant human hyaluronidase PH20 have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with pegylated recombinant human hyaluronidase PH20 in patients with pancreatic cancer were ongoing.

At the time of submission, pegylated recombinant human hyaluronidase PH20 was not authorised anywhere in the EU for pancreatic 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 13 November 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.

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