EU/3/18/2119 - orphan designation for treatment of glioma

Marizomib

Overview

On 14 December 2018, orphan designation (EU/3/18/2119) was granted by the European Commission to Celgene Europe B.V., the Netherlands, for marizomib 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 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 it 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 135,000 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).

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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 517,400,000 (Eurostat 2018).

What treatments are available?

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

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with glioma. Early studies showed that patients with an aggressive form of glioma called glioblastoma multiforme and whose disease progressed despite previous treatment had improvements with this medicine or their disease did not get worse. 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?

In glioma, overactive proteins called proteasomes prevent the cancer cells from dying normally and allow the cancer to grow. Marizomib is expected to block the activity of proteasomes, leading to death of the cancer cells.

What is the stage of development of this medicine?

The effects of marizomib 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 glioma were ongoing.

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

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 November 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.

EU/3/18/2119: Public summary of opinion on orphan designation: Marizomib for the treatment of glioma

AdoptedReference Number: EMA/806418/2018

Key facts

• Active substance:
  Marizomib
• Intended use:
  Treatment of glioma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2119
• Date of designation:
  14/12/2018
• Sponsor:

  Celgene Europe B.V.; Winthontlaan 6 N; 3526 KV Utrecht; The Netherlands; Tel. [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.