EU/3/11/932 - orphan designation for treatment of polycythaemia vera
pegylated proline-interferon alpha-2b (ropeginterferon alfa-2b)
Orphan
Human
Overview
On 9 December 2011, orphan designation (EU/3/11/932) was granted by the European Commission to AOP Orphan Pharmaceuticals AG, Austria, for pegylated proline-interferon alpha-2b for the treatment of polycythaemia vera.
Please note that this product (marketed as Besremi) was withdrawn from the Community Register of designated orphan medicinal products in December 2018 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.
This medicine is now known as ropeginterferon alfa-2b.
Polycythaemia vera is a disease in which the bone marrow (the spongy tissue inside the large bones where blood cells are produced) produces too many red blood cells. This makes the blood thicker and can result in reduced blood flow to the organs and occasionally the formation of blood clots. While some patients with polycythaemia vera do not have any symptoms, others may have itching, tiredness, headache, blurred vision and an enlarged liver and spleen. Patients who develop blood clots in the small blood vessels can also experience a wide range of symptoms including burning pains in the hands. Patients with blood clots in the arteries can have strokes.
Polycythaemia vera is a long-term debilitating and life-threatening condition because it may lead to the formation of blood clots and bleeding, and can result in leukaemia (cancer of the white blood cells) and myelofibrosis (a disease of the bone marrow).
At the time of designation, polycythaemia vera affected approximately 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 152,000 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 506,300,000 (Eurostat 2011).
At the time of designation, hydroxycarbamide and busulfan were authorised in some EU Member States to reduce the number of red blood cells in patients with polycythaemia vera. In addition, phlebotomy (removal of some of the blood from the body) was recommended in some patients to reduce the risk of blood clot formation.
The sponsor has provided sufficient information to show that pegylated proline-interferon alpha-2b might be of significant benefit for patients with polycythaemia vera because early studies show that it might improve the outcome of patients with this condition and be safer than existing treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Pegylated proline-interferon alpha-2b belongs to the group 'interferons'. Interferons are natural substances produced by the body to help it fight against attacks such as viral infections. The exact way alpha interferons work is not fully understood, but it is thought that they act as immunomodulators (substances that modify how the immune system, the body's defence system, works). Interferon-alpha has been used in the management of patients with various blood disorders for more than two decades.
Pegylated proline-interferon alpha-2b is expected to be injected under the skin. The medicine is expected to work in polycythaemia vera by blocking the production of blood cells in the bone marrow.
In this medicine, interferon alfa-2b has been 'pegylated' (coated 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 interferon alfa-2b is produced by a method known as 'recombinant DNA technology': it is made by a bacterium that has received a gene (DNA), which makes it able to produce interferon alfa-2b. The replacement acts in same way as naturally produced interferon alpha.
The effects of pegylated proline-interferon alpha-2b have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with pegylated proline-interferon alpha-2b in patients with polycythaemia vera were ongoing.
At the time of submission, pegylated proline-interferon alpha-2b was not authorised anywhere in the EU for polycythaemia vera 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 7 October 2011 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 proline-interferon alpha-2b (ropeginterferon alfa-2b)
- Intended use
- Treatment of polycythaemia vera
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/11/932
- Date of designation
- Sponsor
AOP Orphan Pharmaceuticals AG
Wilhelminenstrasse 91/IIf
A-1160 Vienna
Austria
Telephone: +43 1 503 72 44 22
Telefax: +43 1 503 72 44 61
office@aoporphan.at
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Besremi at the time of marketing authorisation.
The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the Besremi : Orphan designation withdrawal assessment report (initial 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: