EU/3/16/1680: Orphan designation for the treatment of neuromyelitis optica spectrum disorders

Neuromyelitis optica spectrum disorders are inflammatory disorders that affect mostly the optic (eye) nerve and the spinal cord. They can lead to reduction or loss of vision, loss of sensation, loss of bladder control, weakness and paralysis of the arms and legs.

The disorders occur more frequently in women than in men. They are thought to be caused by the immune system (the body's natural defences) damaging nerve cells.

Neuromyelitis optica spectrum disorders are debilitating and life threatening due to damage to the nervous system function.

At the time of designation, neuromyelitis optica spectrum disorders affected approximately 0.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 21,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

* 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).

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of neuromyelitis optica spectrum disorders. Treatments were aimed at reducing inflammation. They included glucocorticoids, immunosuppressants and plasmapheresis (also called plasma exchange, a procedure to remove certain substances from the liquid part of the blood).

Antibodies against the protein AQP4 are found in most patients with neuromyelitis optica spectrum disorders. AQP4 plays an important role in protecting nerve cells.

The medicine is a monoclonal antibody (a type of protein) that blocks the action of interleukin-6 (IL-6), a protein in the body involved in the production of antibodies against AQP4. By blocking IL-6, the medicine is expected to decrease the production of antibodies against AQP4 and therefore increase the activity of AQP4. This is expected to prevent damage to nerve cells and reduce the symptoms of neuromyelitis optica spectrum disorders.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with neuromyelitis optica spectrum disorders were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for neuromyelitis optica spectrum disorders. Orphan designation of the medicine had been granted in the United States for neuromyelitis optica spectrum disorders.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 May 2016 recommending the granting of this designation.

• 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.