At its meeting of 5 March 2025, the Board of Directors of the Italian Medicines Agency decided to make eligibile for NHS reimbursement 8 medicines (6 different active ingredients), including orphan medicines for rare diseases (1), new chemical molecules (1), generic medicines (4) and extensions of therapeutic indications (2). 

The orphan drug that the NHS will reimburse is Zokinvy (lonafarnib), indicated to reduce the risk of death in people aged one year and older suffering from progeria (or Hutchinson-Gilford syndrome) or from particular progeroid laminopathies. 

Progeria is the rare genetic disease that affected Sammy Basso, the biologist who died on 5 October 2024 after dedicating his life to scientific dissemination and research on the disease. It affects one person for every 4 million live births and has a global prevalence of one person for every 20 million. Around 350 cases are estimated worldwide, 4 of which are reported in Italy.

The new chemical entity that will be eligible for reimbursement is Enrylaze (recombinant chrysantaspase), an antineoplastic for the treatment of acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma (LL) in adult and paediatric patients (from 1 month of age onwards).

The generics eligible for reimbursement are the oral anticoagulant Apixaban Sandoz (apixaban) – which may be marketed at the expense of the NHS upon expiry of the originator patent (Eliquis) – and three generics ofaxitinib (Axitinib Accord, Axitinib Sandoz and Axitinib Teva), an anticancer medicine indicated for the treatment of advanced renal cell carcinoma (RCC) in adult patients.

The extensions of therapeutic indication concern the antiplatelet agent Clopidogrel Alter (clopidogrel) and the antiviral Marivet (glegaprevir and pibrentasvir). Marivet will also be reimbursed for the treatment of chronic hepatitis C virus (HCV) infection in children aged three years and over.