EU/3/10/814 - orphan designation for treatment of von Willebrand disease
Recombinant human von Willebrand factor
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in July 2018 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.
On 26 November 2010, orphan designation (EU/3/10/814) was granted by the European Commission to Baxter Innovations GmbH, Austria, for recombinant human von Willebrand factor for the treatment of von Willebrand disease.
In May 2015, Baxter Innovations GmbH changed name to Baxalta Innovations GmbH.
Von Willebrand disease is a hereditary bleeding disorder caused by the deficiency of von Willebrand factor, a protein in the blood that helps the blood to clot and prevents excessive bleeding.
There are various forms of the disease. Patients with very mild symptoms may be unaware of it. However, those patients with the most severe forms of the disease have excessive and prolonged bleeding that may be life threatening.
At the time of designation, von Willebrand disease affected less than 2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 101,000 people, and is below the threshold 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 506,500,000 (Eurostat 2010).
At the time of designation, the main treatments authorised in the EU for von Willebrand disease were desmopressin (a synthetic hormone that stimulates the body to release more von Willebrand factor) and plasma concentrates containing von Willebrand factor.
The sponsor has provided sufficient information to show that recombinant human von Willebrand factor might be of significant benefit for patients with von Willebrand disease because early studies show that the medicine has a sustained activity over time that may lead to an improved control of bleeding compared with plasma-derived products. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is expected to work by replacing the von Willebrand factor, which is missing in patients with von Willebrand disease. The replacement protein is expected to help control bleeding. The medicine is produced by a method known as 'recombinant DNA technology': it is made by cells that have received a gene (DNA), which makes them able to produce von Willebrand factor.
The effects of recombinant human von Willebrand factor have been evaluated in experimental models.
At the time of submission of the application for orphan designation, a clinical trial with recombinant human von Willebrand factor in patients with von Willebrand disease was ongoing.
At the time of submission, recombinant human von Willebrand factor was not authorised anywhere in the EU for von Willebrand disease 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 9 September 2010 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
- Recombinant human von Willebrand factor
- Intended use
- Treatment of von Willebrand disease
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/10/814
- Date of designation
- Sponsor
Baxalta Innovations GmbH
Industriestrasse 67
A – 1221 Vienna
Austria
Telephone: +43 1 20 100 247 2542
Telefax: +43 1 20 100 547 5990
E-mail: europe_biosci_globalra@baxalta.com
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Veyvondi 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 Veyvondi : Orphan designation withdrawal assessment report (initial authorisation).
Documents related to this orphan designation evaluation
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: