EU/3/22/2717: Orphan designation for the treatment of West syndrome

Tricaprilin induces ketosis when orally ingested. Ketone bodies act as both an energy substrate for neuronal cells and as signalling molecules in multiple pathways. Ketone bodies have effects on mitochondrial stabilization, neurotransmitter systems, gene regulation, and anti-inflammatory activity, which may ultimately produce a positive clinical effect in patients with West syndrome.
Ketone bodies act as an alternative fuel to glucose and can correct energy deficits; the signalling activities include inhibiting histone deacetylases and suppressing inflammasome formation.
Therefore, tricaprilin is expected to reduce spasms, EEG epileptic activity, developmental impairment and progression to other forms of epilepsy in patients with West syndrome.

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 West Syndrome 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, if such a method exists, 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