EU/3/17/1962 - orphan designation for treatment of IgG4-related disease
Humanised Fc-engineered monoclonal antibody against CD19
OrphanHuman
Overview
On 17 January 2018, orphan designation (EU/3/17/1962) was granted by the European Commission to MWB Consulting Ltd, United Kingdom, for humanised Fc-engineered monoclonal antibody against CD19 (also known as XmAb5871) for the treatment of IgG4-related disease.
The sponsorship was transferred to MWB Consulting S.A.R.L., France, in November 2018.
IgG4-related disease is a group of disorders caused by cells of the immune system (the body's natural defences) entering the patient's own tissues and leading to inflammation, swelling and scarring (fibrosis).
The disease comes and goes, and it can affect the pancreas, liver, kidneys, lungs, tear and salivary glands, eyes or any other organ, causing a wide range of symptoms. Patients with the disease often have high levels of an antibody called IgG4 in their blood, and IgG4-producing blood cells have also entered their tissues.
IgG4-related disease is debilitating because of symptoms of organ swelling such as weight loss, fevers, tiredness and pain. It is life threatening because affected organs become damaged and may stop working.
At the time of designation, IgG4-related disease affected less than 3.2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 165,000 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, IgG4-related disease was mainly treated with steroid medicines called glucocorticoids which reduce inflammation.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with IgG4-related disease because data show that the medicine may help patients when glucocorticoids do not work well enough.This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine is a monoclonal antibody (a type of protein) designed to recognise and attach to B cells, which are white blood cells that make antibodies, including IgG4. When the medicine attaches to B cells, it is expected to reduce their activity and so improve symptoms of the disease.
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 IgG4-related disease were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for IgG4-related disease. Orphan designation of the medicine had been granted in the United States for IgG4-related disease.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 December 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
- Humanised Fc-engineered monoclonal antibody against CD19
- Intended use
- Treatment of IgG4-related disease
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1962
- Date of designation
- Sponsor
MWB Consulting S.A.R.L.
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: