EU/3/05/262

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 UV-A and visible lightinduced photosensitivity disorders (chronic actinic dermatitis, cutaneous porphyrias, actinic prurigo and solar urticaria).

What are UV-A and visible light-induced photosensitivity disorders (chronic actinic dermatitis, cutaneous porphyrias, actinic prurigo and solar urticaria)?

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 the UV-A and the 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.

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

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 is equivalent to a total of around 161,000 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 based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

Different methods are used for the treatment of these photosensitivity disorders depending on the nature of the skin disease. Avoidance or minimization 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 antiallergic 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.

How is this medicine expected to work?

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). 

What is the stage of development of this medicine?

The effects of titanium dioxide and bisoctrizole were 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 is 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.

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;
• 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.