EU/3/20/2343 - orphan designation for treatment of anal cancer
retifanlimab
Orphan
Human
Overview
On 19 October 2020, orphan designation EU/3/20/2343 was granted by the European Commission to Incyte Biosciences Distribution B.V, Netherlands, for retifanlimab for the treatment of anal cancer.
Anal cancer is a cancer of the tissues of the anus. Bleeding from the anus is often the first sign of the disease. Other symptoms include pain, discomfort and itching and small lumps around the anus, difficulty controlling bowels (faecal incontinence) and ulcers. Sometimes the cancer causes no symptoms.
Anal cancer is a long-term debilitating disease due to its symptoms such as incontinence, bleeding, itching and non-healing ulcers. This disease is also life threatening and associated with shortened life expectancy.
At the time of designation, anal cancer affected approximately 2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 104,000 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).
*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
At the time of designation, mitomycin was authorised for the treatment of anal cancer in the EU. Other treatments for anal cancer included 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 anal cancer because early studies found that the cancer shrank in patients whose cancer came back after treatment with platinum-based cancer medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Retifanlimab is a monoclonal antibody, a protein that has been designed to block a receptor (‘target’) called PD-1 found on certain cells of the immune system (the body’s natural defences). Some cancers can make a protein that attaches to PD-1 and switches off the immune cells’ ability to attack the cancer. By blocking PD-1, retifanlimab is expected to stop the cancer switching off these immune cells, thereby increasing the immune system’s ability to kill the cancer cells.
The effects of retifanlimab have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with retifanlimab in patients with anal cancer were ongoing.
At the time of submission, retifanlimab was not authorised anywhere in the EU for the treatment of anal cancer. Orphan designation of retifanlimab had been granted in the United States for the treatment of the condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 10 September 2020, 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
- retifanlimab
- Intended use
- Treatment of anal cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2343
- Date of designation
- Sponsor
Incyte Biosciences Distribution B.V.
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: