EU/3/06/367

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Orphan designation

On 11 April 2006, orphan designation (EU/3/06/367) was granted by the European Commission to Kuros Biosurgery International AG, Lichtenstein, for parathyroid hormone (1-34) transglutaminase fusion protein fibrin matrix complex for the treatment of solitary bone cysts.

What are solitary bone cysts?

Solitary bone cysts (also called unicameral or simple bone cysts) are cavities that develop in the interior of bones by no apparent reason. These cavities are called solitary because appear at single localisations, most frequently in areas close to joints of the femur or the upper arm. Solitary bone cysts can either produce symptoms or remain unnoticed. Because of the presence of the solitary cysts, patients bone becomes fragile and spontaneous bone fracture is one of the most common symptoms.

Spontaneous bone fracture can be a repeated problem in some patients and impair bone’s growth. Solitary bone cysts are chronically debilitating in particular due to serious sequelae secondary to bone fracture and bone growth retardation.

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

At the time of designation solitary bone cysts affected less than 1 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 46,000 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?

At the time of submission of the application for orphan designation, no satisfactory method had been authorised in the European Union for treatment of the condition.

How is this medicine expected to work?

Parathyroid hormone is a hormone present in the body that, amongst other functions, stimulates the formation of new bone tissue. Parathyroid hormone (1-34) transglutaminase fusion protein fibrin matrix complex is composed by a part (sequence) of the parathyroid hormone that keeps the hormone activity on bone formation. In the proposed product the hormone sequence is fixed to a support base made of fibrin that will keep the hormone in place and will allow local activity. Fibrin is a natural component of many body tissues and does not have any bone growth stimulating activity by itself. By stimulating the local production of bone the product could induce the filling of the solitary bone cysts.

What is the stage of development of this medicine?

The effects of parathyroid hormone (1-34) transglutaminase fusion protein fibrin matrix complex had been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with solitary bone cysts were initiated.

Parathyroid hormone (1-34) transglutaminase fusion protein fibrin matrix complex was not authorised anywhere worldwide for solitary bone cysts or designated as orphan medicinal product elsewhere for this condition, 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 8 March 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.

Key facts

Product details for <p>Parathyroid hormone (1-34) transglutaminase fusion protein fibrin matrix complex</p>
Active substanceParathyroid hormone (1-34) transglutaminase fusion protein fibrin matrix complex
Medicine Name
Disease/conditionTreatment of solitary bone cysts
Date of decision11/04/2006
OutcomePositive
Orphan decision numberEU/3/06/367

Review of designation

Sponsor’s contact details:

Kuros Biosurgery International AG
C/o Forum Trust reg.
Landstrasse 34
FL-9494 Schaan
Liechtenstein
Telephone: +41 44 200 5614
Telefax: +41 44 200 5714
E-mail: virginia.jamieson@kuros.ch

Patients’ association contact point:

Not available