MONZA (ITALPRESS) – The Italian Medicines Agency (AIFA), on Aug. 21, 2024, approved the reimbursability of emicizumab under the SSN for routine prophylaxis of bleeding episodes in patients with hemophilia A, without factor VIII inhibitors, who have moderate disease with a severe bleeding phenotype.This approval marks an important extension of the indications of emicizumab reimbursed by the SSN to benefit additional patients with hemophilia A. Specifically, emicizumab will also be reimbursed for patients who do not have factor VIII inhibitors and who suffer from a moderate form of the disease, with a severe hemorrhagic phenotype.Hemophilia A is a severe hereditary disorder that affects about 900,000 people worldwide, of whom about 14 percent have a moderate form of the disease. While the treatment and care of those with severe hemophilia A is well established, there is less information and guidance on prophylaxis of moderate hemophilia A. In addition, the severity of hemophilia 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 hemophilia A may not receive appropriate prophylactic treatment and have a worsened clinical burden. Approximately 85 percent of people with moderate hemophilia A report bleeding by a certain age, and one in three have long-term joint symptoms that often require surgery, significantly affecting their quality of life. “The results of the Haven 6 study, have demonstrated the efficacy and safety of emicizumab even in patients with moderate hemophilia A, and this has made it possible to extend the benefits of this treatment to a group of moderate patients who, until now, did not have an equally effective and less invasive treatment option available to them. This advance represents a concrete response to an important unmet clinical need,” said Giancarlo Castaman, Director of the Hemorrhagic Diseases Unit at the AOU Careggi in Florence, Italy. “We are pleased with the positive decision of the regulatory authority on the reimbursability to the NHS of emicizumab for an additional therapeutic indication,” said Anna Maria Porrini, Medical Department Head of Roche Italy. “People with moderate hemophilia A with a severe hemorrhagic phenotype can also have bleeding that can affect their quality of life. We thus renew our commitment to providing therapeutic solutions that can benefit people with hemophilia A in need of treatment. “AIFA’s approval of reimbursability follows EMA’s decision, which is based on results from the phase III HAVEN 6 study, in which emicizumab demonstrated effective bleeding control and a favorable safety profile in people with non-severe hemophilia A without factor VIII inhibitors, for whom prophylaxis was clinically indicated. The decision was also based on data from clinical practice (“real-world”). Emicizumab was evaluated in one of the largest clinical trial programs in hemophilia A subjects with and without factor VIII inhibitors, including eight phase III trials. Today, emicizumab is approved globally as a prophylactic treatment option for people with hemophilia 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 hemophilia A, offering new perspectives and improvements in the quality of life for many patients. The approval of emicizumab in Italy represents a significant breakthrough for the hemophilia community, allowing wider access to innovative treatments and improving long-term disease management. “We welcome AIFA’s decision to extend the reimbursability of emicizumab to patients with moderate hemophilia A and severe hemorrhagic phenotype. This decision opens up new treatment options for those living with this disease even in the moderate form and marks a turning point in improving the quality of life for an increasingly large segment of our patients,” concludes Cristina Cassone, president of the Federation of Hemophilic Associations (FedEmo).-photo press office Havas PR-(ITALPRESS)
