EU/3/13/1170 - orphan designation for treatment of acromegaly

Octreotide acetate

Overview

On 5 August 2013, orphan designation (EU/3/13/1170) was granted by the European Commission to Larode Ltd, United Kingdom, for octreotide acetate (oral use) for treatment of acromegaly.

The sponsorship was transferred to FGK Representative Service GmbH, Germany, in January 2019.

The sponsorship was transferred to Amryt Pharmaceuticals Designated Activity Company, Ireland in February 2022.

The medicinal product has been authorised in the EU as Mycapssa since 2 December 2022. 

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, leading to excess growth of body tissues and organs. Acromegaly usually affects adults in middle age. In over 90% of patients, it is caused by a benign (non-cancerous) tumour of the pituitary gland called a pituitary adenoma. The most common signs of the disease include large hands and feet, enlarged nose, lips and tongue, coarse skin with skin tags and joint aches. 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 (breathing) systems.

Acromegaly is a long-term debilitating disease because of the abnormal growth it causes in tissues and organs in the body.

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

At the time of designation, acromegaly affected less than 2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 102,000 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. At the time of designation, this represented a population of 512,200,000 (Eurostat 2013).

What treatments are available?

At the time of designation, several medicines were authorised in the EU to treat acromegaly, including 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). These medicines were available as solutions for injection into a muscle or under the skin. Other treatments included surgery and, in rare cases, radiotherapy (treatment with radiation).

The sponsor has provided sufficient information to show that octreotide acetate (oral use) might be of significant benefit for patients with acromegaly because the medicine is a new formulation of octreotide to be taken by mouth, which is expected to be easier to use and not to cause injection-related problems such as pain and local reactions. These assumptions 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?

Octreotide is a synthetic substance that mimics the activity of the natural hormone somatostatin. Like somatostatin, octreotide blocks the release of growth hormone, resulting in the reduction of the symptoms and complications of acromegaly.

Octreotide has been available in 'acetate salt' form for the treatment of acromegaly since the 1980s. This medicine will be formulated as an oily suspension to be taken by mouth.

What is the stage of development of this medicine?

The effects of octreotide acetate (oral use) have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with acromegaly were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for acromegaly. Orphan designation had been granted in the United States for the condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 July 2013 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/3/13/1170: Public summary of opinion on orphan designation: Octreotide acetate (oral use) for treatment of acromegaly

AdoptedReference Number: EMA/COMP/443659/2013 Rev. 1

Key facts

• Active substance:
  Octreotide acetate
• Intended use:
  Treatment of acromegaly
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/13/1170
• Date of designation:
  05/08/2013
• Sponsor:

  Amryt Pharmaceuticals Designated Activity Company;

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Mycapssa at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. 

More information is available in the Mycapssa: EPAR - Orphan maintenance assessment report (initial authorisation).