EU/3/19/2190 : Orphan designation for the treatment of hepatitis D virus infection

Hepatitis D virus infection is an infection of the liver with the hepatitis delta (D) virus. It spreads through contact with blood or other body fluids. The hepatitis D virus 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 with hepatitis B infection alone.

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

At the time of designation, hepatitis D virus infection affected approximately 3.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 197,000 people¹, 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).

¹Disclaimer: 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 518,400,000 (Eurostat 2019).

At the time of application for orphan designation, there were no satisfactory treatments for hepatitis D virus infection in the EU.

This medicine contains a substance called peginterferon lambda-1a. It belongs to a group of proteins called interferons, natural substances produced by the body that have a number of actions including helping fight off viruses. The exact way interferons work is not fully understood, but it is thought that they modify how the immune system (the body’s defence system) works. The interferon in the medicine has been attached to a chemical called polyethylene glycol, which enables it to last longer in the body and so prolongs the interferon’s effects.

The effects of peginterferon lambda-1a have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with peginterferon lambda-1a in patients with hepatitis D virus infection were ongoing.

At the time of submission, peginterferon lambda-1a was not authorised anywhere in the EU for the treatment of hepatitis D virus infection. Orphan designation of peginterferon lambda-1a had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 18 July 2019, 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.