AIFA Approves Four New Medicines for Reimbursement, Including Vamorolone for Duchenne Muscular Dystrophy from Age 4

On April 22, 2026, the Italian Medicines Agency (AIFA) Board of Directors approved reimbursement by the Italian National Health Service (SSN) for four new drugs, including Agamree (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.

This decision marks a significant advancement in access to innovative therapies for DMD, a rare genetic disorder characterized by progressive muscle degeneration and weakness. Agamree, a dissociative steroid, is designed to deliver the anti-inflammatory benefits of traditional corticosteroids while reducing their adverse side effects.

The approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) earlier in 2026 and aligns with the U.S. Food and Drug Administration’s (FDA) 2023 approval of vamorolone for DMD in individuals aged 2 years and older.

Clinical evidence supporting the approval comes from the VISION-DMD study, which evaluated ambulatory boys aged 4 to 7 years across 33 trial sites. The study demonstrated that vamorolone improved time to stand test velocity compared to placebo and showed a favorable safety and tolerability profile relative to prednisone.

In addition to Agamree, AIFA approved reimbursement for Nemluvio (nemolizumab) for dermatitis and prurigo nodularis and two antineoplastic drugs, Trecondi and Welireg. The Board also authorized nine therapeutic indication extensions for existing medications, including Abecma, Calquence, Imfinzi, and Amvuttra, and approved six generic drugs for reimbursement covering antibiotics, smoking cessation, Parkinson’s disease, and epilepsy treatments.

The reimbursement decisions aim to enhance patient access to clinically significant innovations while maintaining sustainability within the Italian National Health Service. AIFA emphasized that all approved therapies met rigorous standards for efficacy, safety, and therapeutic value.

For Duchenne muscular dystrophy specifically, the availability of Agamree through public reimbursement expands treatment options beyond traditional glucocorticoids, offering a mechanism that targets inflammation with a potentially improved safety profile. Long-term data from ongoing studies will continue to inform clinical use and outcomes in broader DMD populations, including older children and adults.

As of April 25, 2026, the approved reimbursements are now active under the SSN, enabling eligible patients across Italy to access these therapies through national health coverage.