Italy Approves First Gene Therapy for Hemophilia B, Expanding Access to 22 New Treatments
Key Insights
Italy's AIFA (search) Board approved reimbursement for Hemgenix (etranacogene dezaparvovec), the first gene therapy for severe and moderately severe hemophilia B (search), offering a one-shot treatment alternative to lifelong factor IX infusions.
The approval includes 22 total medicines across two board meetings, featuring orphan drugs for rare diseases like paroxysmal nocturnal hemoglobinuria (search) and invasive candidiasis (search).
Notable approvals include Fruzaqla for metastatic colorectal cancer (search), Exblifep as the first antibiotic funded through Italy's €100 million Innovation Fund for antimicrobial resistance.
Italy's national health service has approved reimbursement for a groundbreaking gene therapy for hemophilia B (search), marking a significant milestone in the treatment of this rare bleeding disorder. The Italian Medicines Agency (search) (AIFA (search)) Board of Directors approved Hemgenix (etranacogene dezaparvovec) as the first one-shot treatment for severe and moderately severe hemophilia B during its July 29 meeting.
Revolutionary Gene Therapy Approach
Hemophilia B (search) is a rare and potentially fatal hereditary blood clotting disorder that causes bleeding in joints, muscles, and internal organs. Currently, patients must undergo continuous, lifelong infusions of coagulation factor IX to compensate for the congenital deficiency and prevent bleeding episodes.
Etranacogene dezaparvovec represents a paradigm shift in treatment approach. This orphan drug, recognized as innovative, is administered as a single intravenous solution using an adenoviral vector to transport the functional gene for Factor IX production into cells. This enables the liver to produce Factor IX protein and increase its levels in the blood, helping to prevent or reduce bleeding episodes.
Comprehensive Drug Approval Package
The AIFA (search) Board approved reimbursement for 22 medicines across two separate meetings in May and July 2025. The July meeting alone resulted in approval for 11 treatments, including three orphan drugs beyond Hemgenix.
Orphan Drug Approvals
Two additional orphan drugs received approval in July: Rezzayo (rezafungin), an antifungal agent for systemic use indicated for treating invasive candidiasis (search) in adults, and Voydeya (danicopan), a new oral medicine for combination use in adult patients with paroxysmal nocturnal hemoglobinuria (search) (PNH) who have residual hemolytic anemia.
The May meeting had previously approved Fabhalta (iptacopan) for monotherapy treatment of adult patients with paroxysmal nocturnal hemoglobinuria (search) who have hemolytic anemia. PNH is characterized by hemolytic anemia, frequent thrombotic events, and bone marrow failure.
Innovation Fund Milestone
A significant development emerged with the approval of Exblifep (cefepime/enmetazobactam), a combination antibiotic indicated for complicated urinary tract infections and hospital-acquired pneumonia. This represents the first medicine authorized by the AIFA (search) Board to be financed through Italy's new €100 million Innovation Fund specifically targeting antimicrobial resistance.
Cancer Treatment Expansions
The approval package included several oncology treatments. Fruzaqla (fruquintinib) received approval for treating adults with metastatic colorectal cancer (search), while Balversa (erdafitinib) was approved for unresectable or metastatic urothelial carcinoma. The parallel import medicine Cyramza (ramucirumab) was approved for advanced stomach cancer or gastroesophageal junction carcinoma, used either in combination or as monotherapy.
Therapeutic Indication Extensions
Multiple existing medicines received expanded indications. Cosentyx (secukinumab) will now be reimbursed for treating moderate to severe active suppurative hydrosadenitis in adults showing inadequate response to conventional systemic therapy. Xtandi (enzalutamide) received expanded coverage for prostate cancer treatment, while Pirfenidone Teva gained approval for idiopathic pulmonary fibrosis.
The July meeting extended indications for six previously reimbursed medicines: Entresto (sacubitril/valsartan sodium salt complex) for heart failure, immunosuppressant Otezla (apremilast), anticancer medicines Tagrisso (osimertinib) and Xalkori (crizotinib), bone structure medicine Voxzogo (vosoritide), and antiviral Zepatier (elbasvir/grazoprevir).
Emergency and Specialized Care
The approvals also addressed emergency medical needs with Giapreza (angiotensin II), a vasoconstrictor for raising blood pressure to normal levels in adult patients with severely low blood pressure, and Metalyse (tenecteplase), an antithrombotic medicine for acute ischemic stroke (search) treatment in adults.
For migraine management, Vydura (rimegepant) received reimbursement approval, expanding treatment options for this neurological condition.