orphan designation
© Oleg Dudko |

Here, we discover that the European Medicines Agency works to foster scientific excellence in the evaluation and supervision of medicines for the benefit of public and animal health in the European Union

The European Medicines Agency (EMA) facilitates the development of and acces to medicines, evaluates applications for marketing authorisation, monitors the safety of medicines across their lifecycle and provides information to healthcare professionals and patients. A wide range of regulatory mechanisms are employed to achieve these aims, which are continuously reviewed and improved.

The EMA’s work covers a number of areas, including:

  • Support for early access.
  • Scientific advice and protocol assistance.
  • Scientific support for advanced-therapy medicines.
  • Orphan designation of medicines for rare diseases.
  • Scientific guidelines on requirements for the quality, safety and efficacy testing of medicines.

Orphan designation

Around 30 million people living in the European Union (EU) suffer from a rare disease. The EMA plays a central role in facilitating the development and authorisation of medicines for rare diseases, known as “orphan medicines”. The agency provides scientific advice specifically for orphan medicines, called protocol assistance. This allows sponsors to get information about the types of studies needed to demonstrate a medicine’s quality, benefits and risks, and information on its significant benefits.

All designated orphan medicines are assessed for marketing authorised centrally in the EU. This allows companies to make a single application to the EMA, resulting in a single opinion and single decision from the European Commission, which is valid in all Member States.

Authorised orphan medicines benefit from 10 years of protection from market competition with similar medicines with similar indications once they are approved. This protection can be extended by a further two years for medicines that have also complied with an agreed paediatric investigation plan granted at the time of review of the orphan medicine designation.

In March 2017, the European Commission granted orphan designation to UK-based GW Research for cannabidiol for the treatment of Lennox-Gastaut syndrome, a severe form of epilepsy that starts in childhood between two and five years of age. At the time of designation, Lennox-Gastaut syndrome affected approximately two in 10,000 people in the EU, the equivalent of around 103,000 people.

The sponsorship transferred to GW Pharma (International) in the Netherlands in April 2019. Cannabidiol for the treatment of Lennox-Gastaut and Drayet syndrome has been authorised in the EU as Epidyolex since 19 September 2019.

Cannabidiol is extracted from the Cannabis sativa plant. Although the way the medicine works is not clearly understood, cannabidiol is thought to act on targets that play a role in the movement of calcium in the cells which, in turn, is important for the transmission of electrical signals in some nerve cells. As seizures are caused by excessive electrical activity in the brain, altering the movement of calcium is expected to reduce or prevent the seizures in Lennox-Gastaut syndrome.

Cannabidiol is also thought to act on adenosine, a chemical messenger in the brain that plays an important role in suppressing seizures.


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