EU/3/16/1667

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.

What is 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.

What is the estimated number of patients affected by the condition?

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 isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*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).

What treatments are available?

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.

How is this medicine expected to work?

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.

What is the stage of development of this medicine?

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.

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.