For the duration of the trial, Spinraza™ reduced the risk of death or the need for assisted ventilation in infants with type 1 SMA by 47%.
In a press release published on January 13, 2017, Biogen Laboratories and IONIS Pharmaceuticals, presented the final results of the ENDEAR trial that terminated in August 2016, at the annual conference of the British Pediatric Neurological Association in Cambridge, UK (January 11-13, 2017). The results presented included data from 121 infants with proximal spinal amyotrophy linked to SMN1 type 1 included in the trial. They demonstrated that Spinraza™ was well tolerated and showed a statistically significant reduction in the number of deaths or the use of permanent ventilation (primary endpoints of the trial) in infants treated with Spinraza™, compared with placebo. Additional results from the ENDEAR trial will be presented at an upcoming medico-scientific congress.
Considering the significant improvement in motor function found in the 78 participants studied during the interim analysis mid-2016, the ENDEAR trial was terminated sooner than expected. It was followed by an open-label study, the SHINE trial, where all participants received Spinraza™. This also allowed for early access to treatment for SMA type 1 patients, as part of an expanded access program (EAP) in countries where permitted by regulation. In France, this early access is in the form of a nominative Temporary Use Authorization (TUA).