Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2019 on request of the sponsor.
On 10 August 2015, orphan designation (EU/3/15/1545) was granted by the European Commission to Seattle Genetics UK, Limited, United Kingdom, for CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA cross-linking pyrrolobenzodiazepine dimer drug for the treatment of acute myeloid leukaemia.
This medicine is now known as vadastuximab talirine.
The sponsorship was transferred to Seagen Ireland Limited, Ireland, in December 2018.
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 approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 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 growth of the cancer when used together with authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is made up of a cytotoxic (cell-killing) substance that is linked to a type of protein called a monoclonal antibody. The monoclonal antibody has been designed to recognise and attach to CD33 receptors present on the surface of AML cells in about 90% of patients. When the antibody attaches to CD33, the AML cells are expected to absorb the antibody, as well as the cytotoxic substance attached to it. Once inside the cancer cell, the cytotoxic substance is released and is then expected to kill the cell, thereby slowing down the growth of the cancer.
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 AML were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for AML or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 July 2015 recommending the granting of this designation.
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.
Seagen Ireland Limited
One Earlsfort Centre
Lower Hatch Street
Dublin 2
Ireland
Tel. +353 234 00 24
E-mail: information@seagen.com
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: