EU/3/16/1735 - orphan designation for treatment of glioma

zoledronic acid

Overview

On 29 August 2016, orphan designation (EU/3/16/1735) was granted by the European Commission to Laboratorio Italiano Biochimico Farmaceutico Lisapharma S.p.A., Italy, for zoledronic acid 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, vomiting, loss of appetite 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 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 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 513,700,000 (Eurostat 2016).

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) to improve survival. 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 zoledronic acid might be of significant benefit for patients with glioma because laboratory studies indicate that it may lead to significant reductions in tumour growth, compared with authorised 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?

Zoledronic acid belongs to a group of medicines called 'bisphosphonates'. It has been authorised in the EU for several years to treat diseases affecting the bones such as osteoporosis.

In glioma, zoledronic acid is expected to work by blocking the action of an enzyme called farnesyl pyrophosphate synthase, which is involved in cell growth and survival. By blocking this enzyme, zoledronic acid is expected to slow down the growth of the glioma cells and therefore reduce the size of the tumour.

The zoledronic acid in this medicine is contained within tiny particles that transport zoledronic acid into the brain.

What is the stage of development of this medicine?

The effects of zoledronic acid have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with this medicine in patients with glioma had been started.

At the time of submission, zoledronic acid 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 13 July 2016 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/3/16/1735 : Public summary of opinion on orphan designation: Zoledronic acid for the treatment of glioma

Adopted

Key facts

• Active substance:
  zoledronic acid
• Intended use:
  Treatment of glioma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1735
• Date of designation:
  29/08/2016
• Sponsor:

  Laboratorio Italiano Biochimico Farmaceutico Lisapharma S.p.A.; via Licinio, 11; 22036 Erba (CO); Italy; 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.