EU/3/01/074

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Orphan designation

On, 11 December 2001 orphan designation (EU/3/01/074) was granted by the European Commission to PPD Global Ltd., United Kingdom, for halofuginone hydrobromide for the treatment of systemic sclerosis.

What is systemic sclerosis?

Systemic sclerosis (also called scleroderma) is a disease of unknown cause, characterised by the accumulation of collagen leading to fibrosis (scar tissue formation) of various severity in different organs such as the skin, blood vessels, digestive tract, lungs, heart and kidneys.
The condition is chronically debilitating and life threatening, in particular due to lung and kidney damage. The progressive deterioration of the lung function remains life-threatening.

What is the estimated number of patients affected by the condition?

At the time of designation systemic sclerosis affected approximately 0.3 to 1.3 in 10,000 in the European Union (EU) *. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 11,300 to 49,000 people.

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union. This represents a population of 377,000,000 (Eurostat 2001).

What treatments are available?

Several products with anti-inflammatory activity had been authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation. Other products were also authorised in some countries in the Community for the treatment of high blood pressure in the lung vessels (pulmonary hypertension), which is one of the major consequences of the disease.
Satisfactory argumentation has been submitted by the sponsor to justify the assumption that halofuginone hydrobromide might be of potential significant benefit for the treatment of systemic sclerosis. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Halofuginone hydrobromide is a chemical compound. According to the sponsor, it acts as an inhibitor (a molecule which represses or prevents another molecule from engaging in a reaction) on the build-up of one type of collagen (type I). Thus, halofuginone hydrobromide could have a potential influence on the excessive deposition of collagen type I in patients with systemic sclerosis.

What is the stage of development of this medicine?

The effects of halofuginone hydrobromide were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with systemic sclerosis were ongoing.

Halofuginone hydrobromide was not marketed anywhere worldwide for the treatment of systemic sclerosis or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 26 October 2001 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • the seriousness of the condition;
  • the existence of alternative methods of diagnosis, prevention or treatment;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Halofuginone hydrobromide</p>
Active substanceHalofuginone hydrobromide
Medicine Name
Disease/conditionTreatment of systemic sclerosis
Date of decision10/12/2001
OutcomePositive
Orphan decision numberEU/3/01/074

Review of designation

Sponsor’s contact details:

PPD Global Ltd
Granta Park
Great Abington, Cambridge
Cambridgeshire CB1 6GQ
United Kingdom
Telephone: +44 1223 374 450
Telefax: +44 1223 374 137
E-mail: sally.amanuel@europe.ppdi.com

Patients’ associations contact points:

Raynaud's & Scleroderma Association
112 Crewe Road
Alsager
United kingdom
ST7 2JA
Telephone: +44 1270 872 776
Telefax: +44 1270 883 556
E-mail: info@raynauds.org.uk

Association des Sclérodermiques de France
41 Rue du Pont de Fer
28260 Sorel-Moussel
France
Telephone: +33 3 84 25 03 04
E-mail: association.asf@club-internet.fr

Scleroderma Liga e.V.
Kelterstraße 23
76227 Karlsruhe
Germany
Telephone: +49 721 40 48 44
Telefax: +49 721 94 15 515