The AIFA Board of Directors, at its meeting on 10 December 2024, approved the reimbursement by the National Health Service of 9 medicines, including orphan medicines for rare diseases (2), equivalent medicines (1), and extensions of therapeutic indications (6).

Authorised orphan drugs are an immunotherapy indicated for Epstein-Barr virus-positive lymphoproliferative disease and a recombinant human enzyme indicated for treating hyperargininemia. This genetic disease occurs around three years of age, causing the progressive loss of stages of psychomotor development and spasticity in the absence of treatment.

The equivalent of a medicine indicated for the treatment of HIV infection in combination with other antiretroviral medicines has been accepted for reimbursement.

Therapeutic extensions of medicines already reimbursed by the NHS fall within hepatitis C, chronic kidney disease, spinal muscular atrophy (SMA), metastatic colorectal cancer (CRC), idiopathic pulmonary fibrosis (IPF) and epithelial ovarian, fallopian tube or primary, advanced peritoneal cancer.