EU/3/15/1582 - orphan designation for treatment of acute myeloid leukaemia
Magrolimab
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in February 2025 on request of the Sponsor.
On 11 November 2015, orphan designation (EU/3/15/1582) was granted by the European Commission to The Chancellor, Masters and Scholars of the University of Oxford, United Kingdom, for humanised monoclonal antibody of the IgG4 kappa isotype targeting CD47 for the treatment of acute myeloid leukaemia.
The sponsorship was transferred to ICON Clinical Research Limited, Ireland, in June 2016.
The sponsorship was transferred to ICON Clinical Research Limited, Ireland, in June 2016 and subsequently to Gilead Sciences Ireland UC, Ireland, in September 2020.
This medicine is now known as magrolimab.
Acute myeloid leukaemia (AML) is a cancer of the white blood cells (cells that fight against infections). In patients with AML, the bone marrow (the spongy tissue inside the large bones, where blood cells are produced) produces large numbers of abnormal, immature white blood cells. These abnormal cells quickly build up in large numbers in the bone marrow and are found in the blood.
AML is a long-term debilitating and life-threatening disease because these abnormal immature cells take the place of the normal blood cells, causing bleeding episodes, blood clots and reducing the patient's ability to fight infections.
At the time of designation, AML affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 51,000 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 512,900,000 (Eurostat 2015).
Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient's age, symptoms and general state of health. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with AML because early studies in experimental models show that it might reduce the number of leukaemia cells and improve survival. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a protein called CD47. CD47 is found on the surface of many cells and protects them against attacks by the body's immune system. CD47 is found at high levels on the surface of cancer cells. By attaching to CD47, the antibody is expected to block signals that stop certain immune cells from attacking cancer cells, thus subsequently contributing to the destruction of the cancer cells.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with AML had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for AML. Orphan designation of the medicine 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 8 October 2015 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
- Magrolimab
- Intended use
- Treatment of acute myeloid leukaemia
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/15/1582
- Date of designation
- Sponsor
Gilead Sciences Ireland UC
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: