EU/3/12/997

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Orphan designation

On 6 June 2012, orphan designation (EU/3/12/997) was granted by the European Commission to Sirius Regulatory Consulting Limited, United Kingdom, for N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for the treatment of schwannoma.

What is schwannoma?

Schwannomas are benign (non-cancerous) slow-growing tumours of the cells that form a protective sheath around the nerves (called Schwann cells). When a schwannoma grows, it exerts pressure against the nerve, causing damage to it. The symptoms of the disease vary depending on the specific nerve involved. Schwannomas often affect cranial nerves (nerves that emerge from the brain) and cause symptoms such as hearing loss, tinnitus (ringing or buzzing in the ears), problems with balance, headache, numbness, and weakness or paralysis in the face.

Schwannoma is a debilitating disease due to damage to the nervous system. It can also be life threatening due to the risk of the tumour becoming cancerous and the interference with vital body functions such as breathing.

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

At the time of designation, schwannoma affected approximately 0.6 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 30,000 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).

*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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

What treatments are available?

At the time of designation, the main treatment for schwannoma was surgery to remove those tumours which were growing rapidly or causing significant symptoms. Surgery can cure the disease in the majority of cases.

The sponsor has provided sufficient information to show that N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide might be of significant benefit for patients with schwannoma because early studies in experimental models show that it may decrease tumour growth, therefore improving the treatment of patients with this condition, particularly patients whose disease has come back after surgery or for whom surgery is not an option. 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?

This medicine is expected to reduce tumour formation mainly by blocking the activity of histone deacetylases (HDACs), proteins that promote cell division. By blocking HDACs, the medicine is expected to stop cells from dividing and multiplying, leading to cell death. The medicine may also work through other means such as damaging the internal skeleton of the tumour cells and blocking the ability of the cells to repair their DNA.

What is the stage of development of this medicine?

The effects of N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide 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 schwannoma had been started.

At the time of submission, this medicine was not authorised anywhere in the EU for schwannoma. Orphan designation of this medicine had been granted in the United States of America for the treatment of schwannoma of the central nervous system.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 April 2012 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.

Key facts

Product details for <p>N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide</p>
Active substanceN-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide
Medicine Name
Disease/conditionTreatment of schwannoma
Date of decision06/06/2012
OutcomePositive
Orphan decision numberEU/3/12/997

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details:

Sirius Regulatory Consulting Limited
29 Hamblin Meadow
Eddington
Hungerford
Berkshire RG17 0HJ
United Kingdom
Telephone: +44 1488 686449
Telefax: +44 1488 686 449
E-mail: info@siriusregulatory.com

Patients' organisations:

For contact details of patients’ organisations whose activities are targeted at rare diseases see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe.
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.