EU/3/15/1564 - orphan designation for treatment of glioma

Dronabinol

cannabidiol

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in November 2015 on request of the sponsor.

On 9 October 2015, orphan designation (EU/3/15/1564) was granted by the European Commission to GW Research Ltd, United Kingdom, for dronabinol and cannabidiol for the treatment of glioma.

What is glioma?

Glioma is a type of brain tumour that affects the 'glial' cells (the cells that surround and support the nerve cells). Patients with glioma can have severe symptoms, but the types of symptoms experienced depend on where the tumour develops in the brain.

Symptoms can include headaches, nausea (feeling sick), loss of appetite, vomiting, and changes in personality, mood, mental capacity and concentration. About one fifth of patients with glioma have seizures (fits) for months or years before the disease is diagnosed.

Glioma is a long-term debilitating and life-threatening disease because of the severe damage to the brain, and is associated with poor long-term survival.

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

At the time of designation, glioma affected approximately 2.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 133,000 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).

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*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 512,900,000 (Eurostat 2015).

What treatments are available?

At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments included surgery, radiotherapy (treatment with radiation), and chemotherapy (medicines to treat cancer) to improve survival. Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure in the skull and medicines to prevent seizures.

The sponsor has provided sufficient information to show that dronabinol and cannabidiol might be of significant benefit for patients with glioma because studies in experimental models showed that the medicine might reduce the size of the tumour and improve the survival of patients with glioma when given with existing treatments. 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?

Dronabinol and cannabidiol are substances found in the cannabis plant and are thought to act in different and complementary ways on glioma.

Dronabinol is expected to work by blocking the action of a protein complex called mTORC1. This prevents the production of proteins needed for the glioma cells to grow and causes substances called sphingolipids to accumulate in the cell, causing the cell to die.

Cannabidiol is thought to decrease the production of other sets of proteins needed by the cancer to grow and invade other cells (called MMP-2 and MMP-9) as well as to develop new blood vessels to supply it with nutrients (called VEF-F). It may also increase the effect of other medicines used for treating glioma.

What is the stage of development of this medicine?

The effects of dronabinol and cannabidiol have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with dronabinol and cannabidiol in patients with glioma were ongoing.

The combination of dronabinol and cannabidiol was authorised as Sativex in a number of EU Member States for the treatment of multiple sclerosis.

At the time of submission, dronabinol and cannabidiol was not authorised anywhere in the EU for glioma or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 September 2015 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.

EU/3/15/1564: Public summary of opinion on orphan designation: Dronabinol and cannabidiol for the treatment of glioma

AdoptedReference Number: EMA/COMP/607076/2015

Key facts

• Active substance:
  • Dronabinol
  • cannabidiol
• Intended use:
  Treatment of glioma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/15/1564
• Date of designation:
  09/10/2015
• Sponsor:

  GW Research Ltd; Sovereign House; Vision Park; Chivers Way, Histon; Cambridge CB24 9BZ; United Kingdom; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.