EU/3/06/378 - orphan designation for treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects

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Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in November 2006 on request of the sponsor.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/3/06/378) was granted by the European Commission to Insmed Europe Ltd., United Kingdom, for mecasermin rinfabate for the treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects.

What is primary insulin-like growth factor-1 deficiency due to molecular or genetic defects?

Insulin-like growth factor-1 (IGF-1) is a naturally occurring hormone secreted (produced) in the body which, together with growth hormone (GH), plays a central role in stimulating growth of the human body. Deficiency of IGF-1 results in short stature (height) and can be caused by an underlying molecular or genetic defect. There may be molecular defects in the specific structures on the surface of cells (receptors) that GH binds to, resulting in resistance to GH, or signalling defects in the cascade of molecular reactions which induce (stimulate) the biological activity of growth, or defects in expression of the gene that codes for production of IGF-1. In these cases the primary deficiency is circulating IGF-1, with GH levels being normal or elevated. The reason why these molecular or genetic defects occur is unknown.
Primary insulin-like growth factor-1 deficiency due to molecular or genetic defects is a serious and chronically debilitating condition.

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

At the time of designation primary insulin-like growth factor-1 deficiency due to molecular or genetic defects affected less than 2 in 10,000 people in the European Union (EU) *. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 92,000 people.

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

There were no approved treatments for primary insulin-like growth factor-1 deficiency at the time of designation.

How is this medicine expected to work?

Mecasermin is a recombinant (artificially synthesised) human insulin-like growth factor-1. Once administered in patients with primary IGF-1 deficiency, mecasermin is expected to replace the natural IGF-1 in the body, thus compensating for its loss. Thus, mecasermin is expected to stimulate the growth of the body in the same way as IGF-1.

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 in patients with primary insulin-like growth factor-1 deficiency 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;
• and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/06/378: Public summary of positive opinion for orphan designation of mecasermin rinfabate for the treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects

Adopted Reference Number: EMEA/COMP/309707/2006

Key facts

• Active substance:
  Mecasermin rinfabate
• Intended use:
  Treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/06/378
• 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]