EU/3/18/2068: Orphan designation for the treatment of growth hormone deficiency

Growth hormone deficiency is a condition where the patient lacks sufficient growth hormone, which is normally released by the pituitary gland at the base of the brain.

Growth hormone promotes growth during childhood and adolescence, and also affects how the body handles proteins, fat and carbohydrates (sugars). In children, the main signs include failure to grow normally and impaired development of bones and skeletal muscle. In adults, the condition can affect the heart, muscles and bones, alter metabolism and cause mental problems such as anxiety and depression.

Growth hormone deficiency is a long-term debilitating condition that includes decreased bone mass, bone fractures and mental problems. The disease can be life-threatening due to the risk of problems with the heart and blood circulation.

At the time of designation, growth hormone deficiency affected approximately 4.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 243,000 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. This represents a population of 517,400,000 (Eurostat 2018).

At the time of designation, medicines containing recombinant human growth hormone were authorised in the EU to treat growth hormone deficiency. These were given to patients by daily injection.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with growth hormone deficiency. Early studies indicate that the use of somapacitan, which has a long duration of action, may be more convenient for patients than current treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Somapacitan is derived from the human growth hormone and acts in the same way to reduce symptoms of the deficiency. Once injected into the patient, somapacitan attaches to a naturally occurring protein in the blood which makes it remain in the body for longer. Because it can remain in the body for longer, the number of times patients need to take their treatment is reduced and thus somapacitan is expected to be more convenient than other treatments.

The effects of somapacitan have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with somapacitan in patients with growth hormone deficiency were ongoing.

At the time of submission, somapacitan was not authorised anywhere in the EU for growth hormone deficiency 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 19 July 2018 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.