EU/3/16/1738 - orphan designation for treatment of invasive aspergillosis
2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide (also known as F901318)(olorofim)
OrphanHuman
Overview
On 14 October 2016, orphan designation (EU/3/16/1738) was granted by the European Commission to F2G Ltd, United Kingdom, for 2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide (also known as F901318) for the treatment of invasive aspergillosis.
This medicine is now known as olorofim.
The sponsorship was transferred to F2G Biotech GmbH, Austria, in March 2019.
The sponsor’s address was updated in October 2021.
Invasive aspergillosis is an infection caused by species of fungi known as Aspergillus. Spores of these fungi are very widely present in the air, but normally do not cause disease. In some people, usually when the immune system (the body's natural defences) is weakened, the fungus can attack and invade the lungs and spread throughout the body via the bloodstream, damaging other organs and tissues including the heart, kidney, liver, brain, and bones.
Invasive aspergillosis is a life-threatening disease that can be fatal due to damage to the lungs and other organs.
At the time of designation, invasive aspergillosis affected approximately 1.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 87,000 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, several antifungal medicines were authorised in the EU for the treatment of invasive aspergillosis, including amphotericin, caspofungin, isavucononazole, itraconazole, posaconazole and voriconazole.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with invasive aspergillosis, with laboratory studies showing that the medicine may be effective against infections resistant to azole antifungal medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine interferes with the production of pyrimidine, a compound the fungi need to make DNA. It does this by blocking an enzyme called dihydroorotate dehydrogenase (DHODH), which is involved in producing pyrimidine. The resulting lack of pyrimidine is expected to prevent the fungi from multiplying and spreading.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with invasive aspergillosis had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for invasive aspergillosis 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 8 September 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
- 2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide (also known as F901318)(olorofim)
- Intended use
- Treatment of invasive aspergillosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1738
- Date of designation
- Sponsor
Update history
| Date | Update |
|---|---|
| October 2023 | The sponsorship was transferred from F2G Biotech GmbH to Shionogi B.V. |
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: