EU/3/18/2106 - orphan designation for treatment of sudden sensorineural hearing loss

6-fluoro-9-methyl-9H-pyrido[3,4-b]-indole

Overview

On 14 December 2018, orphan designation (EU/3/18/2106) was granted by the European Commission to AudioCure Pharma GmbH, Germany, for 6-fluoro-9-methyl-9H-pyrido[3,4-b]-indole for the treatment of sudden sensorineural hearing loss.

What is sudden sensorineural hearing loss?

Sudden sensorineural hearing loss (SSNHL) is a sudden loss of hearing in one or sometimes both ears, caused by damage to the cells in the inner ear that detect sound and to the nerves that connect them to the brain. The damage can have various causes including exposure to loud noise, stress and infection, or may occur as a side effect of certain medicines. Symptoms may develop over a period of up to 3 days, and can include tinnitus (buzzing or ringing in the ears), a sensation of blockage and vertigo (dizziness).

SSNHL is a long-term debilitating condition because of the often irreversible hearing loss it produces.

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

At the time of designation, SSNHL affected approximately 4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 207,000 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 application, various medicines were authorised in the EU to treat symptoms associated with SSNHL such as tinnitus and vertigo. No medicines were authorised to treat the hearing loss itself.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with SSNHL. Early laboratory studies show that the medicine could improve recovery from hearing loss and protect cells in the ear that detect sound.

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?

The medicine, which is to be injected into the middle ear, is thought to work by protecting cells in the ear that detect sound (hair cells) as well as nerve cells that send signals from the ear to the brain. Although the exact way the medicine works is not fully understood, the medicine is expected to reduce cell death and activate the production of neurotrophins, which are small proteins that can repair damaged nerve cells, and thereby help to restore normal ear function.

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 SSNHL had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for SSNHL 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 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/3/18/2106: Public summary of opinion on orphan designation: 6-fluoro-9-methyl-9H-pyrido[3,4-b]-indole for the treatment of sudden sensorineural hearing loss

Adopted Reference Number: EMA/830798/2018

Key facts

• Active substance:
  6-fluoro-9-methyl-9H-pyrido[3,4-b]-indole
• Intended use:
  Treatment of sudden sensorineural hearing loss
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2106
• Date of designation:
  14/12/2018
• Sponsor:

  AudioCure Pharma GmbH; Schlegelstraße, 9; 10115 Berlin; Germany; Tel. [phone]; E-mail: [e-mail]