Roche's Evrysdi Continues to Benefit Children with Spinal Muscular Atrophy

Roche has announced encouraging five-year results for its spinal muscular atrophy (SMA) treatment, Evrysdi (risdiplam), in children. SMA is a severe and progressive neuromuscular disorder that affects about one in every 10,000 babies. The disease is marked by insufficient levels of the SMN protein, which can lead to significant reductions or losses in physical strength, as well as the ability to walk, eat, or breathe.

Evrysdi, developed by Roche, is a liquid medication that can be administered at home either orally or through a feeding tube. It aims to treat SMA by increasing and maintaining the production of the SMN protein in the central nervous system and peripheral tissues. The FIREFISH study, an open-label extension trial, has been investigating the safety and efficacy of this SMN2 splicing modifier in children with type 1 SMA. This form of the disease is particularly severe, with untreated patients typically not surviving past two years and never being able to sit without support.

After five years of treatment with Evrysdi, the results are promising. A significant 91% of patients were still alive, and 81% were alive without the need for permanent ventilation. Additionally, 59% of the children were able to sit without support for at least 30 seconds. The long-term data, presented at the Cure SMA Research and Clinical Care Meeting, further highlighted that some children had gained even more motor functions. By the end of the fifth year, seven children could stand (three with support and four unaided), and six could walk with support.

Motor function abilities were not only maintained but also continued to be achieved in many of the children treated with Evrysdi. At the five-year mark, a notable 96% of those assessed could swallow, and 80% could feed without the need for a feeding tube.

Levi Garraway, Roche's chief medical officer and head of global product development, emphasized the significance of these findings. He noted that the FIREFISH study has provided extensive insights and data, solidifying Evrysdi as a crucial treatment option that improves the lives of children worldwide who are living with SMA.

Roche is at the forefront of the clinical development of Evrysdi, which has already received approval in over 100 countries. This effort is part of a collaborative endeavor with the SMA Foundation and PTC Therapeutics. Moreover, submissions for Evrysdi are currently under review in an additional 13 countries.