EU/3/18/2009

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Orphan designation

On 16 April 2018, orphan designation (EU/3/18/2009) was granted by the European Commission to SOTIO a.s, Czech Republic, for autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo (also known as DCVAC/OvCa) for the treatment of ovarian cancer.

What is ovarian cancer?

Ovarian cancer is cancer of the ovaries, the two organs in the female reproductive system that produce eggs. Most ovarian cancers occur in women aged over 50 years. Due to the absence of clear symptoms in the early stages of the disease, the majority of women are diagnosed when the cancer has spread to other parts of the body.

Ovarian cancer is a debilitating and life-threatening disease that is associated with poor long-term survival.

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

At the time of designation, ovarian cancer affected approximately 4.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 243,000 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 (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for the treatment of ovarian cancer. The choice of treatment depended mainly on how advanced the disease was. Treatments included surgery and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with ovarian cancer. Preliminary results from studies show that the medicine may increase the length of time patients live without their disease getting worse when added to standard chemotherapy.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine works by stimulating cells of the immune system (the body’s natural defences) to attack cancer cells. It is made from the patient’s own dendritic cells (a type of immune cell), whose job is to stimulate other immune cells to attack foreign bodies. Before being injected into the body, the dendritic cells are grown outside the body and mixed with killed ovarian cancer cells to make them better at stimulating the immune cells to attack the cancer cells.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with ovarian cancer were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for ovarian cancer. Orphan designation had been granted in the United States for ovarian cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 March 2018 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.

Key facts

Product details for <p>autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo</p>
Active substanceautologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision16/04/2018
OutcomePositive
Orphan decision numberEU/3/18/2009

Review of designation

Sponsor’s contact details

SOTIO a.s
Jankovcova 1518/2
Prague 7
170 00
Czech Republic
Tel. +420 224 175 111
E-mail: sotio@sotio.com

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.