AIFA Approves Reimbursement for 7 Drugs, Including Skyclarys for Friedreich’s Ataxia

7 drugs (of which 3 orphans), 2 generics and 6 extensions of therapeutic indications were admitted for reimbursement by the AIFA Board of Directors.

The AIFA board of Directors – in the meeting of 15 December – gave the green light to the reimbursement of Skyclarys (omaveloxolone), an orphan drug for the treatment of Friedreich’s ataxia.
friedreich’s ataxia is a rare genetic disease that mainly affects the central nervous system and the heart. It begins during childhood or adolescence and its progression frequently enough leads to the loss of motor independence by the third decade of life, while cardiac complications represent the main cause of mortality.It is indeed estimated to affect 1 in 20,000-50,000 people worldwide. In Italy, the estimated prevalence is approximately 1.07 cases per 100,000 inhabitants.
Skyclarys, the first drug shown to slow down the progression of the disease, was authorized by the European Medicines Agency (EMA) in February 2024 and included by AIFA, in July 2024, in the list of drugs distributed pursuant to law no. 648/1996, an early access program that allows free provision to eligible patients, wiht the cost entirely supported by the National Health Service.