EU/3/14/1394 - orphan designation for treatment of pancreatic cancer
pegylated recombinant human hyaluronidase PH20(pegvorhyaluronidase alfa)
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in October 2025 on request of the Sponsor.
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.
This medicine is now known as pegvorhyaluronidase alfa.
The sponsorship was transferred to Pharma Gateway AB, Sweden, in February 2019.
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.
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 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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).
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.
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.
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.
- 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
- Active substance
- pegylated recombinant human hyaluronidase PH20(pegvorhyaluronidase alfa)
- Intended use
- Treatment of pancreatic cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/14/1394
- Date of designation
- Sponsor
Pharma Gateway AB
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: