EU/3/06/377 - orphan designation for treatment of growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH

mecasermin rinfabate

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in November 2006 on request of the sponsor.

On 20 June 2006, orphan designation (EU/3/06/377) was granted by the European Commission to Insmed Europe Ltd., United Kingdom, for mecasermin rinfabate for the treatment of patients with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH.

What is the condition?

Growth hormone (GH) is a natural hormone secreted (produced) in the body which, together with insulin-like growth factor-1 (IGF-1), plays a central role in stimulating growth of the human body. Deficiency in GH results in short stature (height) and can be caused by a deletion in the GH gene. These patients are usually treated with recombinant (artificially synthesised) human GH, however, some patients develop antibodies to GH during treatment, resulting in loss of growth response.
This is a serious chronically debilitating condition.

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

At the time of designation the number of patients with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH was considered to be less than 0.01 in 10,000 people in the European Union (EU)*. This is equivalent to a total of less than 460 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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 459,700,000 (Eurostat 2004).

What treatments are available?

There were no approved treatments available for patients with GH gene deletion who have developed neutralizing antibodies to GH at the time the application was made.

How is this medicine expected to work?

Mecasermin rinfabate is recombinant (artificially synthesised) human insulin-like growth factor-1 together with a recombinant form of its main transport protein, human insulin-like growth factor-binding protein-3. When a patient with GH deficiency develops neutralising antibodies, treatment with growth hormone is no longer possible and the patients are in need of an alternative treatment intervention. Mecasermin rinfabate could produce similar effects to GH on body growth.

What is the stage of development of this medicine?

The effects of mecasermin rinfabate were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials including some patients with GH gene deletion who have developed neutralizing antibodies to GH were ongoing.
Mecasermin rinfabate was authorised as an orphan medicinal product in the United States for 'the long-term treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to growth hormone', at the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 16 May 2006 a positive opinion recommending the grant of the above-mentioned 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 Community) 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/3/06/377: Public summary of positive opinion for orphan designation of mecasermin rinfabate for the treatment of patients with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH

Adopted Reference Number: EMEA/COMP/309736/2006

Key facts

• Active substance:
  mecasermin rinfabate
• Intended use:
  Treatment of growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/06/377
• Date of designation:
  20/06/2006
• Sponsor:

  Insmed Europe Ltd.; Compass House; Vision Park; Chivers Way; Histon; Cambridge CB4 9AD,; United Kingdom; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]