EU/3/21/2415: Orphan designation for the treatment of small cell lung cancer

Lefitolimod is a short synthetic DNA molecule folding into a dumbbell-like shape. It does not code for gene expression, but it can bind to and activate a receptor (target) called TLR9 on specialised immune cells. This is expected to trigger a broad anti-tumour immune response enabling patients' immune system to recognise and destroy small cell lung cancer cells.

Based on description provided by sponsor

At the time of submission of the application for orphan designation:

• The effects of the medicine had been evaluated in experimental models.

• Clinical trials with the medicine in patients with small cell lung cancer were ongoing.

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

• scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;

• market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

• it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;

• the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;

• there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

• Orphan designation: Overview

• Rare diseases, orphan medicines: Getting the facts straight