EU/3/01/058

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Orphan designation

On 19 September 2001, orphan designation (EU/3/01/058) was granted by the European Commission to Dompé s.p.a., Italy, for repertaxin l-lysinate salt for the prevention of delayed graft function in organ transplant.

The sponsorship was transferred to Dompé pha.r.ma s.p.a, Italy, in April 2007 and subsequently to Dompé s.p.a., Italy, in February 2010.

What is delayed graft function in organ transplant?

Delayed graft function (DGF) is a complication that occurs in the first few days after the transplant of a solid organ, such as a kidney, when the transplanted organ does not start to work properly. DGF may be caused by events occurring after the restoration of blood flow to the transplanted organ. The damage to the organ caused by the interruption and restoration of blood flow is called ischaemia/reperfusion injury and is associated with an inflammatory reaction, characterised by the invasion of neutrophils (a type of white blood cell) into the transplanted organ. DFG is a life-threatening condition because of the risk of losing the transplanted organ.

What is the estimated number of patients at risk of developing the condition?

At the time of designation, the number of patients at risk of delayed graft function in organ transplant was estimated to be approximately 1 people in 10,000 in the European Union (EU) *. This is equivalent to a total of around 38,000 people, which 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 the 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 based on data from the European Union. This represents a population of 377,000,000 (Eurostat 2001).

What methods of prevention are available?

At the time of submission of application for orphan drug designation there were no satisfactory methods for the prevention of delayed graft function in organ transplant. The preventive measures to reduce delayed graft function in organ transplant consisted of donor management and organ preservation.

How is this medicine expected to work?

Repertaxin l-lysinate salt blocks the activity of of interleukin-8, a type of protein called chemokine that is responsible for the reaction of the immune system (body’s natural defences) to unknown factors. IL-8 is a mediator of an inflammatory response that can occur after restoring blood flow in a transplanted organ and cause DGF. Repertaxin l-lysinate salt is suggested to inhibit the inflammatory reaction of tissue associated with restored blood flow and thus prevent the delayed graft function in organ transplant.

What is the stage of development of this medicine?

The effects of repertaxin l-lysinate salt were evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with delayed graft function in organ transplant were initiated.

Repertaxin l-lysinate salt was not marketed anywhere worldwide for the prevention of delayed graft function in organ transplant 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 18 July 2001 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.

Key facts

Product details for <p>Repertaxin L-lysine salt</p>
Active substanceRepertaxin L-lysine salt
Medicine Name
Disease/conditionPrevention of delayed graft function in organ transplant
Date of decision19/09/2001
OutcomePositive
Orphan decision numberEU/3/01/058

Review of designation

Sponsor’s contact details

Dompé s.p.a.
Via Campo di Pile
67100 L'Aquila
Italy
Telephone: + 39 0862 33 81
Telefax: + 39 0862 318108
E-mail: info@dompe.it

Patient associations’ contact points

None available