EU/3/06/422 - orphan designation for treatment of ovarian cancer
paclitaxel
OrphanHuman
Overview
On 18 December 2006, orphan designation (EU/3/06/422) was granted by the European Commission to Oasmia Pharmaceutical AB, Sweden, for paclitaxel (micellar) for the treatment of 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.
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).
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.
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.
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.
- 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
- Active substance
- paclitaxel
- Intended use
- Treatment of ovarian cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/06/422
- Date of designation
- Sponsor
Oasmia Pharmaceutical AB
Vallongatan 1
SE 753 17 Uppsala
Sweden
Tel. + 46 1850 5440
Fax + 46 1851 0873
E-mail: info@oasmia.com
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Apealea at the time of marketing authorisation.
The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the Apealea : Orphan designation withdrawal assessment report (initial authorisation)
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: