Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in May 2007 on request of the sponsor.
On 9 July 2003, orphan designation (EU/3/03/159) was granted by the European Commission to Dr. Geoffrey Allan, United Kingdom, for recombinant human insulin-like growth factor-I / recombinant human insulin-like growth factor binding protein-3 for the treatment of primary growth hormone insensitivity syndrome (Laron Syndrome).
The sponsorship was transferred to Insmed Europe Ltd, United Kingdom, in December 2006.
Laron Syndrome is a growth disorder. The growth hormone (GH) is a natural hormone secreted in the body and responsible for the maturation and growth of the human body. In order to activate the cascade of molecular reactions which induce the biological activity of growth, the GH needs to bind to specific structures on the surface of cells: the GH receptors. Sometimes these GH receptors are defective and unable to respond to GH, in other words, the receptor is “insensitive” to the hormone, which is the case in patients with Laron syndrome. The reason why these receptor defects occur is unknown. Other mechanisms induced by the link of the GH to its receptor can be defective in Laron syndrome patients, such as insulin-like growth factor (IGF-I) production. IGF-I is actually the factor that stimulates body growth. Once the active insulin-like growth factor-I is released in the blood, it is bound to specific transport proteins, such as insulin-like growth factor binding protein 3 (IGFBP-3) in order to transport IGF-I to the tissues and in fact stimulate body growth. Primary growth hormone insensitivity syndrome (Laron syndrome) is a serious chronically debilitating condition.
At the time of designation, primary growth hormone insensitivity syndrome (Laron syndrome) affected approximately 0.03 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 1,100 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: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.
There were no approved treatments available for Laron syndrome at the time the application was made. However, insulin-like growth factor-I has been used for chronic treatments in patients affected by the condition.
The administration of insulin-like growth factor-I is expected to replace the missing active form of growth factor and thereby could produce similar effects on growth and differentiation. In addition, the administration of the complex of the active growth hormone together with its main transport protein (IGF-I/IGFBP-3) could have some advantages over the administration of IGF-I alone (e.g. increase of the mean time insulin growth factor-I is present in the blood, possible administration of higher doses of IGF-I).
At the time of submission of the application for orphan designation, no clinical trials in patients with primary growth hormone insensitivity syndrome (Laron syndrome) were initiated.
Recombinant human insulin-like growth factor-I / recombinant human insulin-like growth factor binding protein-3 was not marketed anywhere worldwide for the treatment of primary growth hormone insensitivity syndrome (Laron Syndrome), at the time of submission.
Orphan designation of recombinant human insulin-like growth factor-I / recombinant human insulin-like growth factor binding protein-3 was granted in the United States for treatment of growth hormone insensitivity syndrome and treatment of major burns that require hospitalisation.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 June 2003 recommending the granting of this designation.
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.
Insmed Europe Ltd
Compass House
Vision Park,
Chivers Way
Histon
Cambridge CB 4 9AD
United Kingdom
Telephone: + 44 1223 25 77 43
Telefax: + 44 1223 25 78 00
E-mail: information@insmed.com
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: