EU/3/10/752 - orphan designation for treatment of Gaucher disease
velaglucerase alfa
OrphanHuman
Overview
On 6 June 2010, orphan designation (EU/3/10/752) was granted by the European Commission to Shire Pharmaceuticals Ireland Limited, Ireland, for velaglucerase alfa for the treatment of Gaucher disease.
Velaglucerase alfa has been authorised in the EU as Vpriv since 26 August 2016.
Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in August 2022 at the end of the 12-year period of market exclusivity.
Gaucher disease is an inherited disorder that is caused by the lack of an enzyme called glucocerebrosidase. This enzyme normally breaks down a fatty waste product called glucocerebroside. Without the enzyme, glucocerebroside builds up in the body, typically in the liver, spleen and bone marrow. This causes a wide range of symptoms, including anaemia (low red blood cell counts), tiredness, easy bruising and a tendency to bleed, an enlarged spleen and liver, and bone pain and fractures.
Gaucher disease is a long-term, debilitating and life-threatening disease that is associated with a reduced life expectancy if left untreated.
At the time of designation, Gaucher disease affected approximately 0.3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 15,000 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, two medicines, imiglucerase and miglustat, were authorised for the treatment of Gaucher disease in the EU. Imiglucerase is an 'enzyme replacement therapy' that works by replacing the missing enzyme. Miglustat blocks the production of glucocerebroside and is used in patients who cannot receive enzyme replacement therapy.
The sponsor has provided sufficient information to show that velaglucerase alfa might be of significant benefit for patients with Gaucher disease because it may represent an alternative treatment to imiglucerase, should the long-term supply problems that are occurring with this medicine continue or happen again in the future. Velaglucerase alfa might also be less 'immunogenic' than imiglucerase. This means that it might be less likely to trigger the production of antibodies (proteins that are produced in response to a substance, which can reduce the effects of a treatment). These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Velaglucerase alfa is an enzyme replacement therapy that is expected to work by replacing the missing enzyme in Gaucher disease, helping to break down glucocerebroside and stopping it building up in the body. Velaglucerase alfa is produced by a method known as 'recombinant DNA technology': it is made by human cells that have received a gene (DNA), which make them able to produce the enzyme.
The effects of velaglucerase alfa have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with velaglucerase alfa in patients with Gaucher disease were ongoing.
At the time of submission, velaglucerase alfa was authorised and had been granted orphan designation in the United States of America for Gaucher disease.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 March 2010 recommending the granting of this designation.
Update: Velaglucerase alfa (Vpriv) was authorised in the EU on 26 August 2010 for for long-term enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease.
- 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
- velaglucerase alfa
- Medicine name
- Vpriv
- Intended use
- Treatment of Gaucher disease
- Orphan designation status
- Expired
- EU designation number
- EU/3/10/752
- Date of designation
- Sponsor
Takeda Pharmaceuticals International AG Ireland Branch
Review of designation
During its meeting of 7-8 July 2010, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/10/752 for Vpriv (velaglucerase alfa) as an orphan medicinal product for the treatment of Gaucher disease. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other satisfactory methods of treatment. As other satisfactory methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained.
The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.
Update history
| Date | Update |
|---|---|
| September 2021 | The sponsorship was transferred to Takeda Pharmaceuticals International AG Ireland Branch, Ireland in September 2021. |
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: