Following our request to receive important and timely updates on their SMA clinical programme, Roche has informed us that worldwide recruitment has completed for the RAINBOWFISH clinical trial, which investigates the efficacy and safety of risdiplam in infants with pre-symptomatic SMA Type 1. This milestone marks the enrolment of 26 participants from across the globe.
What happens now that recruitment is complete?
Whilst recruitment completion is exciting news, the diligent work of trial participants and researchers is ongoing. All participants will receive risdiplam for 2 years as part of the RAINBOWFISH study. Afterward they may enter an open label extension phase that would continue for at least 3 additional years.
When will RAINBOWFISH study results be available?
Overall primary results of the study are expected in early 2023, after all participants have had the opportunity to complete 12 months of treatment from when they enrolled. However, interim (preliminary) data from the RAINBOWFISH study are already available and have been submitted to the European Medicines Agency (EMA) as part of an application to extend risdiplam’s currently approved indication in Europe for patients younger than 2 months old. This application was accepted by the EMA on 17 January, which means that EMA’s Committee for Medicinal Products for Human Use (CHMP) will now conduct a formal scientific evaluation. A positive decision by the CHMP would expand the approved use of risdiplam to infants with SMA across all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.
Are there other risdiplam clinical studies planned?
MANATEE is now the only Roche-sponsored global clinical trial commencing recruitment in early 2022. MANATEE is a two-part, global Phase 2/3 clinical study, which aims to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody targeting muscle growth in combination with risdiplam, in ambulant (able to walk independently) children with SMA aged between 2 and 10 years.