EU/3/16/1713 - orphan designation for treatment of scedosporiosis
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 29 August 2016, orphan designation (EU/3/16/1713) 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 scedosporiosis.
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.
Scedosporiosis is an infection caused by a group of fungi called Scedosporium species. Infection mainly occurs through inhalation of spores and can then spread within the body. Scedosporiosis is usually seen in patients with a weakened immune (defence) system and it is often resistant to many antifungal medicines.
Scedosporiosis is a life-threatening and long-term debilitating disease because it can spread throughout the body and lead to infections in the bone, heart and brain.
At the time of designation, scedosporiosis affected approximately 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of around 500 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 submission of the application for orphan drug designation, the only product authorised for scedosporiosis was the antifungal medicine voriconazole. Surgery was also used to treat the condition.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with scedosporiosis because laboratory studies showed that it could alleviate fungal infections that are resistant to other antifungal medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is expected to block production of pyrimidine in the fungus that causes scedosporiosis. Because the fungus needs pyrimidine for its genetic material, blocking its production prevents the fungus from multiplying and spreading.
At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.
At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with the condition were planned.
At the time of submission, the medicine was not authorised anywhere in the EU for scedosporiosis 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 July 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 scedosporiosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1713
- Date of designation
- Sponsor
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 |
|---|---|
| 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: