EU/3/21/2468: Orphan designation for the treatment of non-traumatic spontaneous intracerebral haemorrhage

Melatonin is a natural substance produced in the body with a variety of activities. It is involved in controlling sleep patterns, it stimulates the production of many enzymes and also acts as an antioxidant (a substance that helps the body protect itself against damage due to reactive substances containing oxygen).

The exact way in which melatonin works in spontaneous intracerebral haemorrhage is not clearly understood, but it has been shown to reduce signs of DNA damage, inflammation, cell death and damage to the mitochondria (the energy-producing components within cells). Melatonin may also increase the production of the proteins HO-1 and NQO1, which are involved in antioxidant activity, in the brain.

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

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

• No clinical trials with the medicine in patients with non-traumatic spontaneous intracerebral haemorrhage had been started.

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