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Orphan designation

On 18 December 2006, orphan designation (EU/3/06/422) was granted by the European Commission to Oasmia Pharmaceutical AB, Sweden, for imexon for the treatment of ovarian cancer.

What is ovarian cancer?

Tumours that begin in the ovaries are known as ovarian tumours and those with the potential to grow rapidly and infiltrate the surrounding healthy tissues are called ovarian cancers. Due to the absence of symptoms in early stages of the disease the majority of patients are diagnosed when the tumours have spread locally or to distant parts of the body. Ovarian cancer is a life-threatening condition.

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

At the time of designation ovarian cancer affected approximately 2.9 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 133,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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 459,700,000 (Eurostat 2004).

What treatments are available?

Several anticancer medicinal products were authorised for the condition in the Community at the time of submission of the application for orphan designation. Although a significant percentage of patients respond to the initial chemotherapy (using drugs to kill cancer cells), most ovarian cancers grow again and respond moderately or poorly to subsequent chemotherapy.

Paclitaxel (micellar) might be of potential significant benefit for the treatment of ovarian cancer because it causes fewer side-effects than currently used treatments. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain orphan status.

How is this medicine expected to work?

Cells contain small structures (so-called microtubules), which are responsible for the structure of the cell and also help during cell-division. Paclitaxel (micellar) blocks the microtubules in the cancer cells in such a way that the cells cannot divide, leading to the destruction of the cell itself. Paclitaxel (micellar) is a new type of formulation.

What is the stage of development of this medicine?

The evaluation of the effects of paclitaxel (micellar) in experimental models is ongoing.

At the time of submission of the application for orphan designation, clinical trials in patients with ovarian cancer were ongoing.
Paclitaxel (micellar) was not authorised anywhere worldwide for treatment of ovarian cancer 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 9 November 2006 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;
  • 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>Paclitaxel (micellar)</p>
Active substancePaclitaxel (micellar)
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision17/12/2006
Orphan decision numberEU/3/06/422

Review of designation

Sponsor’s contact details

Oasmia Pharmaceutical AB
Vallongatan 1
SE 753 17 Uppsala
Tel. + 46 1850 5440
Fax + 46 1851 0873

Patients' organisations

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.