Roche has released three-year data from its FIREFISH study, including one-year data from the open label extension.
FIREFISH evaluated the efficacy and safety of Evrysdi (risdiplam)in infants aged 1-7 months at the time of enrolment with type 1 spinal muscular atrophy (SMA). The study was in two parts, with Part 1 confirming the dosage and Part 2 evaluating the efficacy and safety of that dosage.
The analysis reinforced the long-term efficacy and safety of Evrysdi, with data also showing an estimated 91% of infants treated with the drug were alive after three years of treatment.
Infants continued to improve or maintain motor functions, notably, the ability to swallow, sit without support, stand with support and walk while holding on, following two and three years of treatment. Without the treatment, children with type 1 SMA are not able to sit without support.
The study also showed overall continued reductions in serious adverse events and hospitalisations over time.
Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein in the central nervous system and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.
“These long-term results in babies treated with Evrysdi are very encouraging, with the vast majority improving or maintaining motor functions after three years. Without treatment, they would typically not survive beyond two years of age,” said Levi Garraway, chief medical officer and head of global product development at Roche.
He added: “Support for the compelling efficacy of Evrysdi continues to grow for a broad range of people, including infants with one of the most severe forms of SMA.”
The drug was granted prime designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the US Food and Drug Administration in 2017.
Evrysdi is administered daily at home in liquid form by mouth or by feeding tube and is currently approved in 79 countries, while also under review in a further 29 countries.