EU/3/16/1667 - orphan designation for treatment of small cell lung cancer
rovalpituzumab tesirine
OrphanHuman
Overview
On 30 May 2016, orphan designation (EU/3/16/1667) was granted by the European Commission to Aceso Biologics Consulting Ltd, United Kingdom, for rovalpituzumab tesirine for the treatment of small cell lung cancer.
The sponsorship was transferred to Abbvie Ltd, United Kingdom, in February 2017 and subsequently to AbbVie Deutschland GmbH & Co. KG, in May 2018.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in January 2021 on request of the Sponsor.
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 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 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 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 rovalpituzumab tesirine might be of significant benefit for patients with small cell lung cancer because early studies indicate that it has improved anticancer effects compared with currently authorised medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Rovalpituzumab tesirine is made up of two components, which are linked together:
- a monoclonal antibody (a type of protein) that has been designed to recognise and attach to delta-like 3 (DLL3), a protein that is found on the surface of the cancerous cells in small cell lung cancer;
- a substance toxic for cells.
When injected into the patient, the medicine is expected to attach to the cancerous cells. The medicine is then expected to release the toxic component inside the cells, causing them to die.
The effects of rovalpituzumab tesirine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with rovalpituzumab tesirine in patients with small cell lung cancer were ongoing.
At the time of submission, rovalpituzumab tesirine was not authorised anywhere in the EU for small cell lung cancer. Orphan designation of the medicine has 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 21 April 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
- rovalpituzumab tesirine
- Intended use
- Treatment of small cell lung cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/16/1667
- Date of designation
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: