EU/3/12/1087: Orphan designation for the treatment of growth hormone deficiency

Growth-hormone deficiency is a disease caused by the pituitary gland (a gland located at the base of the brain) not producing enough growth hormone, a hormone responsible for body growth in children and for the control of the correct composition of fat, muscle and bone in adults. As a result, children fail to grow normally and have delayed tooth development and puberty. In adults, low or absent growth hormone can cause reduced muscle and bone mass, low energy, weight gain, heart problems and psychological symptoms such as anxiety and depression. The causes of growth-hormone deficiency are varied and often unknown.

Growth-hormone deficiency is a long-term debilitating and life-threatening condition particularly because of problems with the heart and bones, and psychological symptoms.

At the time of designation, growth-hormone deficiency affected approximately 4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 204,000 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 509,000,000 (Eurostat 2013).

At the time of designation, several medicines containing growth hormone were authorised in the EU for the treatment of growth-hormone deficiency. These medicines are given by daily injection.

The sponsor has provided sufficient information to show that recombinant modified human growth hormone might be of significant benefit for patients with growth-hormone deficiency because it is expected to be given less often than current treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Recombinant modified human growth hormone is expected to work in the body in the same way as human growth hormone. When injected into the patient, it is expected to replace the missing hormone, thereby correcting the deficiency.

This medicine is produced by a method known as 'recombinant DNA technology': it is made by cells that have received a gene (DNA) that makes them able to produce growth hormone. The growth hormone in this medicine has also been modified to decrease the rate at which it is removed from the body, allowing the medicine to be given less often (possibly once a week or once every other week).

The effects of recombinant modified human growth hormone 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 growth-hormone deficiency were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for growth-hormone deficiency. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 December 2012 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.