EU/3/16/1777 - orphan designation for treatment of smallpox
brincidofovir
OrphanHuman
Overview
On 18 November 2016, orphan designation (EU/3/16/1777) was granted by the European Commission to Chimerix UK Ltd, United Kingdom, for brincidofovir for the treatment of smallpox.
The sponsorship was transferred to Chimerix IRL Limited, Ireland, in April 2019.
Smallpox is a very contagious disease caused by variola virus. Symptoms usually begin with high fever, feeling unwell, headache and backache, followed by development of red spots in the throat and mouth that spread to other parts of the body. The spots then form fluid-filled blisters that scar heavily on healing.
Throughout history, smallpox regularly killed large numbers of people around the world. Following effective vaccination, the disease was declared eradicated in 1980. The main risk of future infections comes from potential bioterrorist attacks or accidents in manipulating live virus stock (two declared live stocks remain in the United States and Russia).
Smallpox is debilitating and life-threatening because it causes scarring of the skin, blindness, excessive bleeding and deformities of the arms and legs and the rate of death is high.
At the time of designation, there were no patients affected by smallpox in the European Union (EU). This 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).
At the time of submission of the application for orphan drug designation, no satisfactory methods were authorised in the EU for the treatment of smallpox.
At the time of submission of the application for orphan drug designation, no satisfactory methods were authorised in the EU for the treatment of smallpox.In order for the smallpox virus to multiply and cause disease, copies of its DNA (genetic material) must be made by an enzyme called DNA polymerase. This medicine is expected to block the DNA polymerase of the virus from making new DNA and so prevent the virus from multiplying.
The effects of brincidofovir have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with brincidofovir in patients with smallpox had been carried out. A clinical trial with brincidofovir in patients infected with other viruses was ongoing.
At the time of submission, brincidofovir was not authorised anywhere in the EU for smallpox 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 6 October 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 smallpox
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1777
- Date of designation
- Sponsor
Emergent Operations Ireland Limited
Update history
| Date | Update |
|---|---|
| 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: