EU/3/07/520

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Orphan designation

On 20 December 2007, orphan designation (EU/3/07/520) was granted by the European Commission to ISA Pharmaceuticals BV, the Netherlands, for human-papilloma-virus-type-16 E6 / E7 synthetic long peptides for the treatment of epithelial neoplasia of the vulva, positive for human papilloma virus.

In November 2011, ISA Pharmaceuticals BV changed name to ISA Therapeutics B.V.

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

What is epithelial neoplasia of the vulva positive for human papilloma virus?

An epithelial neoplasia is when cells lining the outer layers of the body (for instance the skin and inside the mouth, intestines and the genitalia) start dividing and growing in an unusual way. An epithelial neoplasia can manifest itself as a wart or a lump, and in some cases it can develop into cancer, if left untreated. A majority of epithelial neoplasias of the vulva (the outer parts of the female genitalia) are caused by an infection of human papilloma virus type 16. Symptoms of the disease include discomfort, itching, irritation, pain and local bleeding. Epithelial neoplasia of the vulva is chronically debilitating and life-threatening due to the associated symptoms and the high risk of developing invasive cancer.

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

At the time of designation, epithelial neoplasia of the vulva positive for human papilloma virus affected approximately 2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 100,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 498,000,000 (Eurostat 2006).

What treatments are available?

There are no medicinal products authorised for the treatment of the condition in the Community. Depending on how advanced the disease is at diagnosis, some patients can treated surgically with removal of the abnormal tissue.

How is this medicine expected to work?

Human-papilloma-virus-type-16 E6 / E7 synthetic long peptides is a vaccine. A vaccine is a compound that resembles part of a disease-inducing foreign organism, such as a virus or a bacteria, designed to 'teach' the body’s natural defence system (the immune system) to recognise, in this case, human papilloma virus type 16. E6 and E7 are two protein molecules that are found in cells infected by the virus. According to the sponsor, vaccination with this medicinal product will help induce the immune system to naturally destroy neoplastic epithelial cells in the vulva.

What is the stage of development of this medicine?

The effects of human-papilloma-virus-type-16 E6 / E7synthetic long peptides were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with epithelial neoplasia of the vulva positive for human papilloma virus were ongoing.

Human-papilloma-virus-type-16 E6 / E7 synthetic long peptides was not authorised anywhere worldwide for the treatment of epithelial neoplasia of the vulva positive for human papilloma virus nor designated as an 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 Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on 8 November 2007 recommending the granting of the abovementioned 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 Community) 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

Product details for <p>Human-papilloma-virus-type-16 E6 / E7 synthetic long peptides</p>
Active substanceHuman-papilloma-virus-type-16 E6 / E7 synthetic long peptides
Medicine Name
Disease/conditionTreatment of epithelial neoplasia of the vulva positive for human papilloma virus
Date of decision19/12/2007
OutcomePositive
Orphan decision numberEU/3/07/520

Review of designation

Sponsor’s contact details

ISA Therapeutics B.V.
J.H. Oortweg 19
2333 CH Leiden
The Netherlands
Tel. +31 71 3322310
Fax +31 71 3322311
E-mail: info@isa-pharma.com

Patients’ associations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.