EU/3/15/1500 - orphan designation for treatment of hepatitis delta virus infection
OrphanHuman
Overview
This medicine is now known as bulevirtide.
On 19 June 2015, orphan designation (EU/3/15/1500) was granted by the European Commission to MYR GmbH, Germany, for synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus for the treatment of hepatitis delta virus infection.
The sponsor’s address was updated in September 2019.
Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus has been authorised in the EU as Hepcludex since 31 July 2020.
The sponsorship was transferred to Gilead Sciences Ireland Unlimited Company in June 2021.
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.
Key facts
- Medicine name
- Hepcludex
- Intended use
- Treatment of hepatitis delta virus infection
- Orphan designation status
- Positive
- EU designation number
- EU/3/15/1500
- Date of designation
- Sponsor
Gilead Sciences Ireland Unlimited Company
Ida Business Park
Carrigtwohill T45 DP77
Co. Cork
Ireland
Tel: +353 12913504
E-mail: Regulatory.orphan@gilead.com
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Hepcludex at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Hepcludex : Orphan maintenance assessment report.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: