EU/3/11/947 - orphan designation for treatment of acromegaly

recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin serotype D

Overview

On 11 January 2012, orphan designation (EU/3/11/947) was granted by the European Commission to Syntaxin Limited, United Kingdom, for recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin serotype D for the treatment of acromegaly.

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in November 2014 on request of the Sponsor.

What is acromegaly?

Acromegaly is a disease in which the pituitary gland, a small gland located at the base of the brain, produces too much growth hormone. Acromegaly usually affects adults in middle age. In over 90% of patients, it is caused by a non-cancerous tumour of the pituitary gland called a pituitary adenoma. One of the most common symptoms of the disease is the abnormal growth of the hands and feet. The disease can result in serious complications, such as severe damage to the joints and problems affecting the cardiovascular (heart and blood vessels) and respiratory systems.

Acromegaly is a long-term debilitating disease that can be life threatening because of joint damage, risk of cardiovascular disease and reduced life expectancy.

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

At the time of designation, acromegaly affected approximately 1.3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of 66,000 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, several medicines were authorised in the EU to treat acromegaly, including bromocriptine and somatostatin analogues (medicines that block the release of growth hormone) such as octreotide and lanreotide, and pegvisomant (a medicine that blocks the effects of growth hormone). Other treatments include surgery and, in rare cases, radiotherapy (treatment with radiations).

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with acromegaly because it works in a different way to existing treatments and early studies show that it might improve the treatment of patients with this condition. 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 consists of 'growth hormone releasing hormone' (GHRH) which has been linked to an enzyme called endopeptidase (which is derived from a bacterial toxin called botulinum toxin). The medicine is expected to block the secretion of growth hormone from the pituitary gland. It is expected to do this by attaching to GHRH receptors located on the surface of cells in the pituitary gland, and interfering with a protein which enables the release of growth hormone from the pituitary cells.

The medicine is expected to be given into a vein.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

At the time of submission, no clinical trials with the medicine in patients with acromegaly had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for acromegaly 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 9 November 2011 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/3/11/947: Public summary of positive opinion for orphan designation: Recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin...

Adopted Reference Number: EMA/COMP/928881/2011

Key facts

• Active substance:
  recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin serotype D
• Intended use:
  Treatment of acromegaly
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/11/947
• Date of designation:
  11/01/2012
• Sponsor:

  Syntaxin Limited; Units 4-10 The Quadrant; Barton Lane; Abingdon OX14 3YS; United Kingdom; Telephone: [phone]; Telefax: [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.