Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in September 2011 on request of the Sponsor.

On 5 December 2001, orphan designation (EU/3/01/073) was granted by the European Commission to NeuTec Pharma plc, United Kingdom, for recombinant human monoclonal antibody to hsp90 for the treatment of invasive fungal infections.

The sponsorship was transferred to Novartis Europharm Limited, in June 2007.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Invasive fungal infections are serious infections from various types of fungal species. The most common invasive fungal infections include invasive candidosis, aspergillosis, and cryptococcosis.

Invasive candidosis, also known as systemic candidosis or candidiasis, is an infection of visceral organs (large interior organs in the body cavities such as abdomen) by yeasts of the genus Candida. It encompasses infections that range from superficial to systemic. While superficial infections of skin and mucosal membranes, such as oral or vaginal thrush, cause local inflammation and discomfort, the systemic infections are potentially life threatening, especially in severely immunocompromised persons where the infection can spread through the blood stream to multiple organs.

Invasive aspergillosis is an acute infection caused by the micro-organism Aspergillus. This infection affects mainly patients with a weak immune system, including patients with AIDS or neutropenia (abnormally low number of a type of white blood cell called a neutrophil). A rapidly invasive aspergillus infection in the lungs often causes cough, fever, chest pain, and difficulty in breathing. Aspergillosis affecting the deeper tissues makes a person very ill. Symptoms include fever, chills, shock, and blood clots. Patients may develop kidney and liver failure.

Cryptococcosis is another serious and potentially fatal fungal disease which similar to other invasive fungal infections, affects mostly people with a defective immune system. Cryptococcosis is due to Cryptococcous neoformas. The symptoms include chest pain, dry cough, swelling of the abdomen, headache, blurred vision, and confusion. Invasive fungal infections are life threatening.

At the time of designation, invasive fungal infections affected 0.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 11,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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. At the time of designation, this represented a population of 378,800,000 (Eurostat 2001).

Several anti-fungal medicinal products were authorised for the condition in the Community at the time of submission of the application for orphan designation.

Recombinant human monoclonal antibody to hsp90 might be of potential significant benefit for the treatment of invasive fungal infections, because it could improve the treatment of invasive fungal infections. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

Hsp90 is a protein synthesized by the microorganisms in response to stress. It is thought to play role in fungal infections. Recombinant human monoclonal antibody to hsp90 is made by a cell that has received a gene (DNA) that makes it able to produce a protein called hsp90 (heat shock protein 90). Antibodies are proteins naturally found in the blood that help the body to fight infections and other diseases. The recombinant human monoclonal antibody to hsp90 is expected to recognise the hsp90 produced by the fungi, and to bind to it, thus triggering the body's immune system to attack and lower levels of fungi causing infection.

The effects of recombinant human monoclonal antibody to hsp90 were evaluated in experimental models. At the time of submission of the application for orphan designation, a clinical trial in patients with invasive fungal infections was ongoing.

Recombinant human monoclonal antibody to hsp90 was not marketed anywhere worldwide for the treatment of invasive fungal infections 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 7 September 2001 recommending the granting of this designation.

  • 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.

EU/3/01/073: Public summary of positive opinion for orphan designation of recombinant human monoclonal antibody to hsp90 for the treatment of invasive fungal infections

Key facts

Active substance
Recombinant human monoclonal antibody to hsp90
Intended use
Treatment of invasive fungal infections
Orphan designation status
Withdrawn
EU designation number
EU/3/01/073
Date of designation
Sponsor

Novartis Europharm Limited
Frimley Business Park
Camberley GU16 7SR
United Kingdom
Tel. +41 61 324 11 11 (Switzerland)
E-mail: orphan.enquiries@novartis.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:

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