EU/3/16/1697 - orphan designation for treatment of adenovirus infection in immunocompromised patients
brincidofovir
OrphanHuman
Overview
On 14 July 2016, orphan designation (EU/3/16/1697) was granted by the European Commission to Chimerix UK Ltd, UK, for brincidofovir for the treatment of adenovirus infection in immunocompromised patients.
The sponsorship was transferred to Chimerix IRL Limited, Ireland, in April 2019.
Adenovirus is a common virus that causes a wide range of illnesses such as colds, sore throat and eye infection. Infection with adenoviruses is rarely severe in healthy people but it can lead to life-threatening infections of lung, liver, gut, bladder and brain in immunocompromised patients (patients with weakened immune systems). These include patients who have had haematopoietic (blood) stem cell transplantation or organ transplantation and those with diseases that weaken the immune system, including AIDS.
Adenovirus infection in immunocompromised patients is life threatening and debilitating in the long term due to the spread of severe infection.
At the time of designation, adenovirus infection in immunocompromised patients affected approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 154,000 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 513,700,000 (Eurostat 2016).
At the time of designation, no satisfactory methods were authorised for treatment of adenovirus infection in the EU.
Brincidofovir is an antiviral medicine that belongs to the class 'nucleotide analogues'. It blocks the activity of enzymes called 'DNA polymerases' in adenovirus, which the virus uses to produce DNA. When the virus cannot produce DNA, it cannot reproduce, slowing down the spread of infection.
The effects of brincidofovir 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 adenovirus infection in immunocompromised patients were ongoing.
At the time of submission, brincidofovir was not authorised anywhere in the EU for adenovirus infection in immunocompromised patients 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 16 June 2016 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
- Active substance
- brincidofovir
- Intended use
- Treatment of adenovirus infection in immunocompromised patients
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1697
- Date of designation
- Sponsor
Symbio Pharma Ireland Limited
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| March 2024 | The sponsorship was transferred from Emergent Operations Ireland Limited to Symbio Pharma Ireland Limited in March 2024. |
| August 2023 | The sponsorship was transferred from Chimerix IRL Limited, Ireland to Emergent Operations Ireland Limited, Ireland in August 2023. |
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: