EU/3/19/2143: Orphan designation for the treatment of small cell lung cancer

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 1.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 73,000 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 518,400,000 (Eurostat 2019).

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 lurbinectedin might be of significant benefit for patients with small cell lung cancer, with early studies suggesting that this medicine may lead to better outcomes for patients than are seen with existing treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Lurbinectedin is expected to work by breaking down an enzyme called ‘RNA polymerase II’, which plays a key role in the production of proteins that are needed for the cell to grow and multiply. Small cell lung cancer cells have high levels of these proteins, which make the cells grow uncontrollably. By breaking down RNA polymerase II, lurbinectedin reduces production of these growth-related proteins and so reduces the growth of the cancer.

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 24 January 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.