EU/3/20/2272 - orphan designation for treatment of myasthenia gravis
Rozanolixizumab
OrphanHuman
Overview
On 22 April 2020, orphan designation EU/3/20/2272 was granted by the European Commission to UCB Pharma, Belgium, for rozanolixizumab for the treatment of myasthenia gravis.
Myasthenia gravis is a disease that leads to muscle weakness and tiredness. It is an autoimmune disorder in which the immune system (the body’s natural defences) attacks and damages muscle cells, making them unable to contract normally. For a muscle to contract, a substance called acetylcholine is released from a nerve and attaches to the acetylcholine receptors on the muscle cells. In myasthenia gravis, an immune system protein called IgG antibody triggers the immune system to damage these receptors and the muscles are not able to contract as well as normal.
In most patients, the disease is associated with abnormalities of a gland in the chest called the thymus, which is part of the immune system.
The muscles involved in swallowing and those around the eyes are commonly affected first, causing difficulty in swallowing and the eyelids to drop. Muscle weakness typically worsens towards the end of the day and after exercise.
Myasthenia gravis is a long-term debilitating disease and may be life-threatening when the muscles involved in breathing are affected.
At the time of designation, myasthenia gravis affected approximately 2 in 10,000 people in the European Union (EU). This was equivalent to a total of 104,000 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).
* 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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
At the time of designation, several medicines were authorised in the EU for the treatment of myasthenia gravis, including acetylcholinesterase inhibitors (medicines that prevent breakdown of acetylcholine) and medicines that work on the immune system. Surgery to remove the thymus gland (thymectomy) was performed in some patients.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with myasthenia gravis because early data showed that rozanolixizumab improved the symptoms of the disease in patients who were not adequately controlled on standard treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Rozanolixizumab is a monoclonal antibody (a type of protein) designed to recognise and attach to FcRn, a protein that keeps the IgG antibodies in the body for longer. By binding to and blocking FcRn, the medicine increases the removal of these antibodies, preventing them from triggering the attack on the acetylcholine receptors. This is expected to lead to an improvement in muscle function.
The effects of rozanolixizumab have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with rozanolixizumab in patients with myasthenia gravis were ongoing.
At the time of submission, rozanolixizumab was not authorised anywhere in the EU for the treatment of myasthenia gravis. Orphan designation of rozanolixizumab had been granted in the EU for the treatment of immune thrombocytopenia and in the USA for primary immune thrombocytopenic purpura and for myasthenia gravis.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 19 March 2020, 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
- Rozanolixizumab
- Intended use
- Treatment of myasthenia gravis
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2272
- Date of designation
- Sponsor
UCB Pharma
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing 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: