EU/3/18/2097 - orphan designation for treatment of glioma

Larotrectinib

Overview

Please note that this product (marketed as Vitrakvi) was withdrawn from the Community Register of designated orphan medicinal products in July 2019 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.

 

On 19 November 2018, orphan designation (EU/3/18/2097) was granted by the European Commission to Bayer AG, Germany, for larotrectinib 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 radiotherapy (treatment with radiation), chemotherapy (medicines to treat cancer) and surgery. 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 larotrectinib might be of significant benefit for patients with glioma because early data showed that the condition did not get worse in patients in whom authorised treatments were not effective. 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?

Some patients with glioma have genetic mutations (changes) called NTRK gene fusions which result in the production of altered TRK proteins that can cause cancer.

Larotrectinib blocks the activity of the altered TRK proteins, thus preventing or slowing down the growth of glioma.

What is the stage of development of this medicine?

The effects of larotrectinib 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, larotrectinib was not authorised anywhere in the EU for glioma. Orphan designation of the medicine had been granted in the United States for soft tissue sarcoma, solid tumors with NTRK-fusion proteins and infantile fibrosarcoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 October 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/3/18/2097: Public summary of opinion on orphan designation: Larotrectinib for the treatment of glioma

AdoptedReference Number: EMA/776178/2018

Key facts

• Active substance:
  Larotrectinib
• Intended use:
  Treatment of glioma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/18/2097
• Date of designation:
  19/11/2018
• Sponsor:

  Bayer AG; 51368 Leverkusen; Germany; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Vitrakvi 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 Vitrakvi : Orphan designation withdrawal assessment report (initial authorisation).