EU/3/13/1220

On 16 January 2014, orphan designation (EU/3/13/1220) was granted by the European Commission to Coté Orphan Consulting UK Limited, United Kingdom, for poly[2-[(4-{[1-carboxy-2-(hexadecylcarbamoyl)ethyl]sulfanyl}-2,3-bis({2-[((2S)-2-(2-{[(2R)-2-carbamoyl-(2-{[(2S)-1-ethoxy-3-(3-hydroxy-4oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}ethyl]sulfanyl}-3-{[(2S)-1-ethoxy-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}propanamido)-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)propanoyl ethyl ester))-methoxy]acetyl}oxy)butyl)sulfanyl]-3-(hexadecylcarbamoyl)propanoic acid]-PEG1500-ester] for the treatment of dengue.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is dengue?

Dengue is a viral infection that is transmitted by the bite of infected mosquitoes. Shortly after being bitten, patients can develop fever together with severe headache, vomiting, and muscle and joint pains. In a small fraction of patients, the disease may progress and cause abnormalities of blood clotting, bleeding and internal leakage of fluid from blood vessels, shock (inadequate blood supply to the organs) and organ failure.

Dengue is potentially life-threatening because of the leakage from the blood vessels and bleeding caused by severe infection.

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

At the time of designation, dengue affected less than 0.1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 5,000 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: 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 512,200,000 (Eurostat 2013).

What treatments are available?

At the time of submission of the application for orphan designation, no satisfactory methods were authorised in the EU for the treatment of dengue.

How is this medicine expected to work?

The medicine is composed of a substance that is designed to attach specifically to the dengue virus and which is distributed along a long molecule called a polymer. This arrangement of the substance along the polymer allows the medicine to wrap round the virus after which it is thought to strip away the virus’s outer coat, making the virus inactive and thereby helping to stop the progression of the disease.

What is the stage of development of this medicine?

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

At the time of submission of the application for orphan designation, no clinical trials with this medicine in patients with dengue had been started.

At the time of submission, this medicine was not authorised anywhere in the EU for dengue. Orphan designation of the medicine had been granted in the United States for treatment of dengue fever (including dengue haemorrhagic fever and dengue shock syndrome).

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 November 2013 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.