EU/3/15/1549

On 9 October 2015, orphan designation (EU/3/15/1549) was granted by the European Commission to Apeptico Forschung und Entwicklung GmbH, Austria, for synthetic peptide L-cysteine, L-cysteinylglycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-, cyclic (1.fwdarw.17)-disulfide for the treatment of primary graft dysfunction following lung transplantation.

What is primary graft dysfunction following lung transplantation?

Organ transplantation refers to the transfer of organs from a donor’s to a recipient’s body. The transplanted organ is called a graft. Transplanted organs may suffer damage during transplantation; in some cases, the damage stops the graft from working normally.

The graft may stop working properly in the first 72 hours after transplantation and this is known as primary graft dysfunction. It is thought that interruption and resumption of blood supply during transplantation can damage the transplanted lungs.

Primary graft dysfunction following lung transplantation is a life-threatening condition because the transplanted organ may fail.

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

At the time of designation, primary graft dysfunction following lung transplantation affected approximately 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of around 500 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 512,900,000 (Eurostat 2015).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of primary graft dysfunction following lung transplantation.

How is this medicine expected to work?

During transplantation, interruption and resumption of blood supply to transplanted lungs can lead to the formation of reactive oxygen species (ROS, toxic molecules containing oxygen) which can damage the cells and the function of the transplanted lungs.

Although its mechanism of action is not fully understood, this medicine is thought to reduce the production of ROS and thereby prevent cells in the transplanted lung from being damaged. It is also thought to restore the impaired exchange of oxygen between the blood and airways in the lung.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, a clinical trial with the medicine in patients with primary graft dysfunction following lung transplantation had been completed.

At the time of submission, the medicine was not authorised anywhere in the EU for primary graft dysfunction following lung transplantation or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 September 2015 recommending the granting of this 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 EU) 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.

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.