EU/3/17/1966 - orphan designation for treatment of small cell lung cancer
N-(bromoacetyl)-3,3-dinitroazetidine
OrphanHuman
Overview
On 17 January 2018, orphan designation (EU/3/17/1966) was granted by the European Commission to Sirius Regulatory Consulting Limited, United Kingdom, for N-(bromoacetyl)-3,3-dinitroazetidine (also known as RRx-001) for the treatment of small cell lung cancer.
The sponsorship was transferred to Sirius Regulatory Consulting EU Limited, Ireland, in October 2018.
Small cell lung cancer is a type of lung cancer that usually develops in the central part of the lungs, and in which the cancer cells are small compared with other types of lung cancer. Small cell lung cancer is almost always caused by smoking. The cancer is difficult to detect in the early stages of the disease, and the majority of the patients are diagnosed when the cancer has spread and cannot be removed by surgery.
Small cell lung cancer is a life-threatening disease that is associated with poor long-term survival.
At the time of designation, small cell lung cancer affected approximately 1.2 in 10,000 persons in the European Union (EU). This was equivalent to a total of around 62,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 515,700,000 (Eurostat 2017).
At the time of designation, several medicines were authorised in the EU for the treatment of small cell lung cancer. The choice of treatment depended mainly on how advanced the disease was. Treatments included chemotherapy (medicines to treat cancer) and radiotherapy (treatment with radiation).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with small cell lung cancer, with early studies suggesting that this medicine may improve the effectiveness of platinum-based chemotherapy. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Although it is not known exactly how this medicine works, it is thought that it works mainly by encouraging macrophages (cells of the immune (defence) system) to attack cancer cells and protecting normal cells from the toxic effects of radio- and chemotherapy.
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 small cell lung cancer were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for small cell lung cancer. Orphan designation of the medicine had been granted in the United States for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 December 2017 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
- N-(bromoacetyl)-3,3-dinitroazetidine
- Intended use
- Treatment of small cell lung cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1966
- Date of designation
- Sponsor
Sirius Regulatory Consulting EU Limited
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: