EU/3/17/1856 - orphan designation for treatment of neuromyelitis optica spectrum disorders
Inebilizumab
OrphanHuman
Overview
On 20 March 2017, orphan designation (EU/3/17/1856) was granted by the European Commission to AstraZeneca AB, Sweden, for inebilizumab (also known as MEDI-551) for the treatment of neuromyelitis optica spectrum disorders.
The sponsorship was transferred to Quality Regulatory Clinical Ireland Ltd, Ireland, in September 2018.
The sponsorship was transferred Viela Bio B.V., Netherlands in November 2020.
Please note that this product (marketed as Uplizna) was withdrawn from the Union Register of orphan medicinal products by the European Commission in March 2022 at the time of the granting of a marketing authorisation.
Neuromyelitis optica spectrum disorders are inflammatory disorders that mainly affect 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) mistakenly producing antibodies that attack the 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 isbased 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 515,700,000 (Eurostat 2017).
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 medicines such as corticosteroids and immunosuppressants, as well as plasmapheresis (also called plasma exchange, a procedure to remove antibodies from the liquid part of the blood).
Inebilizumab is a monoclonal antibody (a type of protein) that attaches to a type of immune cell called CD19+ B cells and causes their death. CD19+ B cells produce antibodies responsible for attacking the nerves in neuromyelitis optica spectrum disorders. By reducing the numbers of B cells, the medicine is expected to prevent damage to nerve cells and reduce the symptoms of the condition.
The effects of inebilizumab have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with inebilizumab in patients with neuromyelitis optica spectrum disorders were ongoing.
At the time of submission, inebilizumab was not authorised anywhere in the EU for neuromyelitis optica spectrum disorders. Orphan designation of inebilizumab had been granted in the United States for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 February 2017 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.
Key facts
- Active substance
- Inebilizumab
- Intended use
- Treatment of neuromyelitis optica spectrum disorders
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1856
- Date of designation
- Sponsor
Viela Bio B.V.
Schiphol Boulevard 359
1118 BJ Schiphol
Netherlands
Email: info@vielabio.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
The Committee for Orphan Medicinal Products reviewed the orphan designation of Uplizna at the time of marketing authorisation, and recommended that the orphan designation should not be maintained.
More information is available in the Uplizna : Orphan maintenance assessment report (initial authorisation).
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: