EU/3/18/2041 - orphan designation for treatment of behavioural variant frontotemporal dementia

2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA

Overview

On 31 July 2018, orphan designation (EU/3/18/2041) was granted by the European Commission to Ionis USA Ltd, United Kingdom, for 2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA for the treatment of behavioural variant frontotemporal dementia.

The sponsorship was transferred to Biogen Netherlands B.V., The Netherlands in October 2020.

What is behavioural variant frontotemporal dementia?

Behavioural variant frontotemporal dementia is a brain disorder in which patients gradually lose their ability to control or adjust their behaviour in different situations, leading to inappropriate behaviour. Language skills may also be affected, with the patient not being able to speak correctly, to pronounce words properly or to remember the right words.

The exact cause of the disease is unclear, but it is thought to be related to the abnormal build-up and tangling of a protein in the brain called tau, which leads to damage in different areas of the brain. The parts of the brain that are affected are the frontal and temporal lobes (front and sides of the brain).

Behavioural variant frontotemporal dementia is a debilitating disease that can be life threatening because of its damaging effects on the brain.

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

At the time of designation, behavioural variant frontotemporal dementia affected approximately 2.6 in 10,000 people in the European Union (EU). This was equivalent to a total of 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?

No satisfactory methods of treatment were authorised in the EU for behavioural variant frontotemporal dementia at the time of designation. Patients were managed with the help from experts such as psychologists, physiotherapists and speech therapists.

How is this medicine expected to work?

The medicine is an antisense oligonucleotide, a type of genetic material that interferes with the genetic material in cells and stops them from producing the tau protein.

By reducing levels of tau in the brain, the medicine is expected to reduce brain damage and help relieve patients’ symptoms.

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, no clinical trials with the medicine in patients with behavioural variant frontotemporal dementia had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for behavioural variant frontotemporal dementia 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 21 June 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/3/18/2041: Public summary of opinion on orphan designation: 2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA for the treatment of behavioural variant frontotemporal dementia

Adopted Reference Number: EMA/470356/2018

Key facts

• Active substance:
  2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA
• Intended use:
  treatment of behavioural variant frontotemporal dementia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2041
• Date of designation:
  31/07/2018
• Sponsor:

  Biogen Netherlands B.V.