The National Health Service will reimburse the first one-shot treatment for severe and moderately severe haemophilia B, a rare and potentially fatal disease that causes bleeding in the joints, muscles and internal organs.

At its meeting on 29 July, the AIFA Board of Directors gave the green light to the reimbursability of Hemgenix (etranacogene dezaparvovec), the first gene therapy authorised for haemophilia B, which can have a significant impact on patients' quality of life. Currently, people with this hereditary blood clotting disorder must undergo continuous, lifelong infusions of coagulation factor IX to compensate for the congenital deficiency and prevent bleeding. Etranacogene dezaparvovec, an orphan drug that has been recognised as innovative, is administered in a single intravenous solution and uses an adenoviral vector to transport the functional gene for Factor IX production into cells. This allows the liver to produce Factor IX protein and increase its levels in the blood, helping to prevent or reduce bleeding episodes.

A total of 11 treatments will be reimbursed by the NHS. These include two other orphan drugs:

• Rezzayo (rezafungin), an antifungal agent for systemic use indicated for the treatment of invasive candidiasis in adults;
• Voydeya (danicopan), a new oral medicine to be used in combination in adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.

Two new chemical molecules will be eligible for reimbursement: Balversa (erdafitinib), an anticancer medicine indicated for unresectable or metastatic urothelial carcinoma, and Exblifep (cefepime/enmetazobactam), a combination antibiotic indicated for complicated urinary tract infections and hospital-acquired pneumonia, the first authorised by the AIFA Board of Directors to be financed through the new €100 million Innovation Fund for medicines targeting antimicrobial resistance.

The extensions of therapeutic indications concern six medicines already reimbursed for other indications: the heart failure medicine Entresto (sacubitril/valsartan sodium salt complex), the immunosuppressant Otezla (apremilast), the anticancer medicines Tagrisso (osimertinib) and Xalkori (crizotinib), the medicine that acts on bone structure and mineralisation Voxzogo (vosoritide), and the antiviral Zepatier (elbasvir/grazoprevir).