Roche's Evrysdi improves survival, motor function in children with SMA: 5-year data

Five years of treatment with Roche Korea's spinal muscular atrophy (SMA) drug Evrysdi (risdiplam) in pediatric patients with type 1 SMA showed significant improvements in survival and the ability to walk, stand, and sit without assistance.

The long-term clinical data for Evrysdi is significant because, without treatment, children with type 1 SMA are not only unlikely to reach developmental milestones such as walking and standing, but are also more likely to die before reaching the age of two. 

Roche Korea said on Friday that five-year long-term clinical data from Evrysdi confirmed the continued efficacy and safety of the treatment in pediatric patients with type 1 SMA.

The results from the open-label extension of FIREFISH, the pivotal phase 3 study of Evrysdi, were presented at the Cure SMA Research & Clinical Care Meeting from June 5-7 in Austin, Texas, the U.S.

According to published data, after five years of treatment with Evrysdi, patients' survival rates and ability to sit, stand, and walk unassisted were maintained and improved. 

After five years of Evrysdi treatment, 91 percent of pediatric patients were alive, 81 percent were free of permanent respiratory support, and 59 percent were able to sit unassisted for more than 30 seconds. 

Of the seven patients who were able to stand, three were able to stand with assistance, four were able to stand without assistance, and six were able to walk with assistance. 

Evrysdi patients maintained or gained motor function as assessed by the Bayley Scales of Infant and Toddler Development (BSID-III) and Hammersmith Infant Neurological Examination (HINE-2) scales for gross motor skills. 

FIREFISH results showed that Evrysdi maintained the ability to eat and swallow in a majority of patients, with 96 percent maintaining swallowing ability and 80 percent being able to eat without a feeding tube at five years of treatment.

No patients discontinued treatment or withdrew from the study due to treatment-related adverse events, and the rate of adverse events decreased by 66 percent from year 1 to year 5. The most common adverse events were upper respiratory tract infections (64 percent), fever (64 percent), and pneumonia (50 percent). Hospitalization rates decreased over the study period, with 22 percent of patients not requiring hospitalization at all after starting Evrysdi treatment.

"The results of this long-term study confirm the sustained effectiveness of Evrysdi in children with type 1 SMA," said Dr. Giovanni Barranello at the University College London Great Ormond Street Institute of Child Health & Great Ormond Street Hospital.

"Over five years of Evrysdi treatment, patients were able to maintain and improve their ability to sit, stand and walk, which are important functions of daily living and growth, and the majority of patients maintained their ability to swallow and feed without a feeding tube."

The final analysis of the FIREFISH study provides meaningful data that confirms the importance of Evrysdi in helping to improve the lives of children with spinal muscular atrophy around the world, said Dr. Levi Garraway, Head of Global Product Development and Chief Medical Officer at Roche. 

Evrysdi is the only oral, non-invasive, small molecule treatment for SMA that works systemically, including the central nervous system and periphery. Roche is leading the clinical development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics.