Hemophilia A, Aifa extends reimbursement for bispecific antibody

The Italian Medicines Agency (AIFA) has recently extended the reimbursement of emicizumab by the National Health Service. The bispecific antibody directed at factor IXa and factor X, already approved for severe haemophilia A with and without factor VIII inhibitors, will now also provide a practical and effective prophylactic treatment option for people with moderate haemophilia A (FVIII≥1% and ≤5%) with a severe bleeding phenotype.

Haemophilia A is a serious inherited disease that affects approximately 900,000 people worldwide, of whom approximately 14% have a moderate form of the disease. While the treatment and care of people with severe haemophilia A is well established, there is less information and guidance on prophylaxis for moderate haemophilia A. Furthermore, the severity of haemophilia A – traditionally measured by an individual’s factor VIII levels – does not always adequately reflect the risk of bleeding. As a result, many people with moderate haemophilia A may not receive appropriate prophylactic treatments and may experience a worsened clinical burden. Approximately 85% of people with moderate haemophilia A report bleeding by a certain age, and one in 3 people with moderate haemophilia A experience long-term joint symptoms that often require surgery, significantly impacting their quality of life.

“The results of the registration study “Haven 6 – explains Giancarlo Castaman, director of the Hemorrhagic Diseases Unit at Aou Careggi in Florence – have demonstrated the efficacy and safety of emicizumab also in patients with moderate hemophilia A, and this has allowed us to extend the benefits of this treatment to a group of patients with moderate form who, until now, could not have an equally effective and less invasive therapeutic option. This progress represents a concrete response to an important clinical need that is still unmet”. In this regard, “we are pleased with the positive decision of the regulatory authority on the reimbursement by the National Health System of emicizumab for an additional therapeutic indication – says Anna Maria Porrini, Medical Department Head of Roche Italy – Even people affected by moderate hemophilia A with a severe bleeding phenotype can have bleeding that can affect their quality of life. We thus renew our commitment to providing therapeutic solutions that can be useful to people with hemophilia A who need treatment”.

The approval of the reimbursement of Aifa follows the decision of the European Agency (EMA) which is based on the results of the phase III Haven 6 study, during which emicizumab demonstrated effective control of bleeding and a favourable safety profile in subjects with non-severe haemophilia A, without factor VIII inhibitors, for whom prophylaxis was clinically indicated. The decision was also based on data from clinical practice (real-world). Emicizumab has been evaluated in one of the largest clinical study programmes in subjects with haemophilia A with and without factor VIII inhibitors, including 8 phase III studies. Today, emicizumab is approved globally as a prophylactic treatment option for people with haemophilia A with factor VIII inhibitors in more than 110 countries and for people without factor VIII inhibitors in more than 100 countries.

Its approval marked a major advance in the management of haemophilia A, offering new perspectives and improvements in the quality of life of many patients. The approval of emicizumab in Italy represents a significant turning point for the haemophilia community, allowing broader access to innovative treatments and improving long-term disease management.

“We welcome with satisfaction the decision of AIFA to extend the reimbursement of emicizumab also to patients with moderate haemophilia A and severe haemorrhagic phenotype – concludes Cristina Cassone, president of the Federation of haemophiliac associations (FedEmo) – This decision opens up new treatment options for those living with this disease even in its moderate form and marks a turning point in improving the quality of life for an increasingly large segment of our patients”.