Approvals

Santhera Pharmaceuticals has received positive final guidance from the National Institute for Care and Excellence (NICE) for AGAMREE (vamorolone) to be used as treatment of Duchenne Muscular Dystophy (DMD) in patients who are aged 4 or older in the UK.

The guidance follows the approval of vamorolone from the Medicines and products Regulatory Agency (MHRA) in January earlier this year. AGAMREE is currently the only fully approved treatment for DMD in the UK, EU and US. Approval from the European Medicines Agency (EMA) was given in late 2023, following on from the approval from the US Food and Medicines Agency (FDA). EMA approval was based on the results of a clinical trial, VISION-DMD, which demonstrated a reduction in adverse events related to bone growth in comparison to prednisolone, another type of corticosteroid.

At the time of the FDA approval, CEO of Santhera Dario Eklund stated: “We are delighted to secure FDA approval, which comes just weeks after the positive opinion from the CHMP of the EMA. This is a hugely important moment for DMD patients who need an efficacious and well-tolerated therapy for this debilitating condition.”

Duchenne Muscular Dystrophy is an orphan disease that primarily affects young boys due to the way it is inherited. Children affected by the disease may have difficulty walking, sitting, running, speaking and developing due to muscle weakness.