EU/3/13/1170: Orphan designation for the treatment of 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.

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 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 28), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 512,200,000 (Eurostat 2013).

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.

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.

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.

• 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.