EU/3/15/1500: Orphan designation for the treatment of hepatitis delta virus infection

Hepatitis delta virus infection (hepatitis D) is an infection of the liver with the hepatitis delta virus. The hepatitis delta virus is known as an 'incomplete' virus, because it cannot replicate in cells without the help of another virus, the hepatitis B virus. Because of this, the virus is always present as an additional infection in patients with hepatitis B, and results in higher rates of liver failure and cirrhosis (scarring of the liver) than is seen with hepatitis B infection alone.

Hepatitis delta virus infection is life threatening and debilitating in the long term as it can lead to cirrhosis, liver failure and portal hypertension (high blood pressure in the vessels that connect the liver and the gut).

At the time of designation, hepatitis delta virus infection affected approximately 4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 205,000 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 512,900,000 (Eurostat 2015).

At the time designation, there were no satisfactory treatments for hepatitis delta virus infection in the EU.

This medicine is made from a protein found in the outer envelope around the hepatitis B virus. It is expected to work by attaching to, and blocking, a receptor through which the hepatitis B and hepatitis delta viruses enter liver cells. By blocking the entry of hepatitis delta virus into cells, the medicine limits its ability to replicate and its effects in the body, so reducing symptoms of infection.

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

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with hepatitis delta virus infection were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for hepatitis delta virus infection 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 13 May 2015 recommending the granting of this designation.

• 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.