EU/3/15/1437 - orphan designation for treatment of acute myeloid leukaemia
alvocidib
OrphanHuman
Overview
On 12 February 2015, orphan designation (EU/3/15/1437) was granted by the European Commission to Theorem Clinical Research GmbH, Germany, for alvocidib for the treatment of acute myeloid leukaemia.
In February 2018, Theorem Clinical Research GmbH changed name to Chiltern Clinical Research International GmbH.
In July 2019, Chiltern Clinical Research International GmbH.changed name to Covance Clinical Development.
In March 2022, the sponsor’s address was updated and the name was changed from Covance Clinical Development GmbH to Labcorp Clinical Development GmbH.
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 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 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 alvocidib might be of significant benefit for patients with AML because initial studies suggest that it can improve responses when added to authorised treatments, including in patients whose disease has come back after previous treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Alvocidib is a medicine that blocks the action of enzymes in the body called 'cyclin-dependent kinases' that are involved in the control of cell division (needed to produce new cells), as well as in the production of new proteins. By blocking the action of these enzymes, alvocidib can prevent leukaemia cells from dividing, eventually resulting in the death of the cell. In addition, if given before other cancer treatments that act at a particular stage of cell division, it can synchronise the cancerous cells so that they are in the most vulnerable stage at time other cancer treatments are given, maximising the effect of treatment.
The effects of alvocidib have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with alvocidib in patients with AML were ongoing.
At the time of submission, alvocidib was not authorised anywhere in the EU for AML. Orphan designation of alvocidib had been granted in the United States for treatment of this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 January 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
- alvocidib
- Intended use
- Treatment of acute myeloid leukaemia
- Orphan designation status
- Positive
- EU designation number
- EU/3/15/1437
- Date of designation
- Sponsor
Labcorp Clinical Development GmbH
Carl-Wery-Strasse 42
Ramersdorf-Perlach
Munich
Bavaria
81739
Germany
E-mail: Evanthia.Fritz@labcorp.com
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: