EU/3/19/2213: 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 of growth hormone deficiency 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 in 10,000 people in the European Union (EU). This was equivalent to a total of around 207,000 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 518,400,000 (Eurostat 2019).

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 clinical data indicate that the use of lonapegsomatropin leads to faster growth than existing treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
 

Lonapegsomatropin is a ‘prodrug’ of somatropin (human growth hormone), meaning that it is converted to somatropin in the body. The somatropin in the medicine is attached to polyethylene glycol (PEG), with a linker that attaches the two. The linker is expected to break down gradually in the blood to release the somatropin over a long period. In this way the somatropin is expected to remain in the body for longer and it can be injected less frequently than other growth hormone medicines.

The effects of lonapegsomatropin have been evaluated in experimental models.

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

At the time of submission, lonapegsomatropin 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, the COMP adopted a positive opinion on 12 September 2019, 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.