EU/3/05/262 - orphan designation for treatment of ultraviolet-A- and visible-light-induced photosensitivity disorders
titanium dioxide
Bisoctrizole
OrphanHuman
Overview
Please note that this product was withdrawn from the Community register of designated orphan medicinal products in July 2013 on request of the sponsor.
On 3 March 2005, orphan designation (EU/3/05/262) was granted by the European Commission to Orfagen, France, for titanium dioxide and bisoctrizole for the treatment of ultraviolet (UV)-A- and visible-light-induced photosensitivity disorders (chronic actinic dermatitis, cutaneous porphyrias, actinic prurigo and solar urticaria).
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
UV-A- and visible-light-induced photosensitivity disorders include various skin diseases, which have in common that exposure to UV rays or even to visible light results in an abnormal skin reaction (photosensitivity). This can produce skin lesions such as redness, scratches, blisters and scarring. Among the skin diseases induced by UV-A and visible light, chronic actinic dermatitis, actinic prurigo and solar urticaria appear without an underlying cause, while cutaneous porphyria occurs in people having an underlying metabolic disease.
At the time of designation, UV-A- and visible-light-induced photosensitivity disorders affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 163,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 466,600,000 (Eurostat 2005).
Different methods are used for the treatment of these photosensitivity disorders depending on the nature of the skin disease. Avoidance or minimisation of light exposure remains the current main treatment for all these disorders. At the time of submission of the application for orphan-drug designation, additional treatments such as anti-inflammatory, emollients and anti-allergic products were commonly used. Also phototherapy (exposure of the skin to a specific light regimen), plasmapheresis (the washout of certain harmful substances from plasma) and phlebotomy (removal of small amounts of blood from the veins) were used in specific situations. Titanium dioxide and bisoctrizole might be of potential significant benefit for the treatment of the above-specified photosensitivity disorders. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.
Titanium dioxide and bisoctrizole cream is expected to combine a high sun-blocking protection (UV-B and UV-A range) together with an extended protection to normal visible light. To obtain this maximum photoprotection, the cream contains a mineral compound (titanium dioxide) and a chemical filter (bisoctrizole).
The effects of titanium dioxide and bisoctrizole have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with UV-A- and visible-light-induced photosensitivity disorders were ongoing.
Titanium dioxide and bisoctrizole was not marketed anywhere worldwide for the treatment of UV-A- and visible-light-induced photosensitivity disorders (chronic actinic dermatitis, cutaneous porphyrias, actinic prurigo and solar urticaria) or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 January 2005 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
- titanium dioxide
- Bisoctrizole
- Intended use
- Treatment of ultraviolet-A- and visible-light-induced photosensitivity disorders
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/05/262
- Date of designation
- Sponsor
Orfagen
CRDPF Langlade
3 Avenue Hubert Curien - BP 13562
31035 Toulouse Cedex 1
France
Tel. +33 5 34 50 64 58
Fax +33 5 34 50 34 57
E-mail info@orfagen.com
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: