Alhemo - withdrawal of application for variation to marketing authorisation
Application withdrawn
The application for a change to this medicine's authorisation has been withdrawn
concizumab
Post-authorisationHuman
Alhemo is a medicine used to prevent bleeding episodes in people aged 12 years and older with haemophilia A or B. People with haemophilia A or B are at risk of bleeding because they lack proteins that help the blood clot (factor VIII for haemophilia A and factor IX for haemophilia B).
Alhemo is used:
- in people with haemophilia A or B who have developed antibodies (proteins made by the body’s natural defences) called inhibitors that stop factor VIII or factor IX medicines from working properly;
- in people who have severe haemophilia A or moderate to severe haemophilia B and have not developed inhibitors.
Alhemo contains the active substance concizumab and is given by injection under the skin of the abdomen (belly) or thigh once a day, using a pre-filled pen. It has been authorised in the EU since December 2024.
The company applied to extend the use of Alhemo to prevent bleeding episodes in children below 12 years of age with haemophilia A or haemophilia B, with or without factor VIII or IX inhibitors.
People with haemophilia A or B have low levels of clotting factor VIII or IX, which makes it harder for their blood to clot and stop bleeding. In addition to factor VIII and IX, the body also uses other proteins to help the blood clot, such as factor Xa.
The active substance in Alhemo, concizumab, is a monoclonal antibody (a type of protein). It attaches to a protein called tissue factor pathway inhibitor (TFPI). TFPI normally reduces the activity of factor Xa and slows down blood clotting. By blocking TFPI, concizumab helps the blood to clot and prevent bleeding in people with haemophilia A or B.
The company presented partial data from an ongoing study which was to involve at least 127 children below 12 years of age with haemophilia A or B, with or without inhibitors. At the time of submission, data from a group of 42 children, all with inhibitors, were presented. Most of the children involved had not previously received Alhemo; however, 10 had received Alhemo as part of a compassionate use programme before entering the study.
At the start of the study, the children had been receiving on-demand or preventive treatment for bleeding episodes for the last year. The main measure of effectiveness was the number of spontaneous or traumatic (due to an injury) bleeding episodes being treated while using Alhemo.
The application was withdrawn after the European Medicines Agency had evaluated the initial information from the company and had prepared questions. The Agency was awaiting the company’s responses to the questions at the time of the withdrawal.
Based on the review of the information, at the time of the withdrawal, the Agency had concerns and its provisional opinion was that, with the information provided at that stage, Alhemo could not have been authorised to prevent bleeding episodes in children below 12 years of age with haemophilia A or haemophilia B, with or without inhibitors.
The company submitted efficacy results from a subgroup of 30 children, all with inhibitors, who had completed the study. Key safety and efficacy data were missing, including efficacy data for children without inhibitors and those younger than 2 years of age. Inconsistencies and lack of clarity about how data were being collected and when the remaining data would be available also made it difficult to interpret the results.
Therefore, at the time of the withdrawal, the Agency’s opinion was that the company had not provided enough information to support the application for a change to the marketing authorisation of Alhemo.
In its letter notifying the Agency of the withdrawal of application, the company stated that it withdrew its application because the Agency considered that the data provided would not support a recommendation for all the proposed indications.
The company informed the Agency that there are no consequences for patients in clinical trials or in compassionate use programmes using Alhemo.
If you are in a clinical trial or compassionate use programme and need more information about your treatment, speak with your clinical trial doctor.
There are no consequences for the use of Alhemo in its authorised uses.