EU/3/13/1179 - orphan designation for treatment of growth hormone deficiency

recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains (somavaratan)

Overview

On 5 August 2013, orphan designation (EU/3/13/1179) was granted by the European Commission to Larode Ltd, United Kingdom, for recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains for the treatment of growth-hormone deficiency.

This medicine is now known as somavaratan.

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in December 2018 on request of the Sponsor.

 

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. The condition can be caused by a genetic mutation 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 functioning of the heart, and cause reduced ability to perform exercise and 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 in 10,000 people in the European Union (EU). This was equivalent to a total of 205,000 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).

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*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).

What treatments are available?

At the time of designation, several medicines containing recombinant human growth hormone were already 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 recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains might be of significant benefit for patients with growth-hormone deficiency because early studies show that it may act for longer in the body, which would allow it to be injected less frequently than existing medicines, thereby making it easier for patients to take their treatment as intended.

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?

The medicine 'recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains' is expected to act in the same way as existing treatments for growth-hormone deficiency, by replacing the missing growth hormone in the patient's body.

The medicine is made of growth hormone attached to two chains of amino acids. It is produced by a method known as 'recombinant DNA technology': it is made by bacteria into which a gene (DNA) has been introduced that makes them able to produce the growth hormone along with the two chains of amino acids. The two chains of amino acids are expected to prolong the time needed for the medicine to be broken down in the body.

What is the stage of development of this medicine?

The effects of recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains 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 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 11 July 2013 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/3/13/1179: Public summary of opinion on orphan designation: Recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains for the treatment of growth-hormone deficiency

Adopted Reference Number: EMA/COMP/440859/2013 Rev. 1

Key facts

• Active substance:
  recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains (somavaratan)
• Intended use:
  Treatment of growth hormone deficiency
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/13/1179
• Date of designation:
  05/08/2013
• Sponsor:

  Larode Ltd; Rhode Farm; Priory Lane; Selborne; Hampshire GU34 3BU; United Kingdom; 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.