EU/3/18/2035 - orphan designation for treatment of growth hormone deficiency

Efpegsomatropin

Overview

On 27 June 2018, orphan designation (EU/3/18/2035) was granted by the European Commission to Hanmi Europe Limited, United Kingdom, for efpegsomatropin for the treatment of growth hormone deficiency.

The sponsorship was transferred to JVM Europe B.V., Netherlands, in April 2019.

What is growth hormone deficiency?

Growth hormone deficiency is a condition where the patient lacks a sufficient amount of growth hormone, which is normally secreted by the pituitary gland (at the base of the brain). Growth hormone promotes growth during childhood and adolescence, and also acts on the way the body handles proteins, fat and carbohydrates (sugars).

The condition can be caused by a genetic mutation (change) or other factors such as trauma and inflammation, or it may have no known cause. It can affect people of any age. In childhood, the main signs include failure to grow normally and impaired development of bones and skeletal muscle. In adulthood, the condition can affect the heart, muscles and bones, and cause psychological symptoms such as anxiety and depression.

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

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

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).

What treatments are available?

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 results in patients with the condition indicate that the medicine has a more prolonged effect than existing treatments and may improve patient outcomes. This assumption 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?

Efpegsomatropin is a modified version of synthetic human growth hormone. Normal growth hormone is quickly broken down in the body, which makes its effects short-lived. In efpegsomatropin, the growth hormone has been attached to a part of another protein, IgG4, which is found in the blood and has a longer life in the body. This helps protect the medicine from being broken down quickly, and allows the growth hormone to have a prolonged action over a week or more.

What is the stage of development of this medicine?

The effects of efpegsomatropin 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, efpegsomatropin 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 24 May 2018 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/3/18/2035: Public summary of opinion on orphan designation: Efpegsomatropin for the treatment of growth hormone deficiency

Reference Number: EMA/380699/2018

Key facts

• Active substance:
  Efpegsomatropin
• Intended use:
  Treatment of growth hormone deficiency
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2035
• Date of designation:
  27/06/2018
• Sponsor:

  JVM Europe B.V.; Niels Bohrweg 6; Lelystad; Flevoland; 8218 MD; The Netherlands; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.