sIn early August 2016, Biogen and Ionis Pharmaceuticals announced that their investigational drug for the treatment of spinal muscular atrophy (SMA), nusinersen has met the pre-specified primary endpoint for the interim analysis of ENDEAR (A study testing the clinical efficacy and safety of IONIS_SMN Rx in patients with infantile-onset SMA). The observed effect of nusinersen on infants with infantile-onset (consistent with type 1) SMA was a statistically significant improvement in the achievement of motor milestones in comparison to infants who had not received the treatment. Nusinersen’s safety profile was also deemed acceptable during the study. The results obtained in the pre-specified interim analysis have led to the closure of the ENDEAR study, thus transitioning participants into the SHINE open-label study in which all patients will be receive nusinersen.
In light of this news, Biogen will develop and commercialize nusinersen globally and will process regulatory filings in the coming months. The executive vice president and chief medical officer at Biogen, Alfred Sandrock, M.D., Ph.D., highlighted “we share the community’s sense of urgency as we strive to bring the first treatment of SMA, the leading genetic cause of infant mortality, to families facing this devastating disease. We remain committed to understanding the potential of nusinersen in the broader SMA population and will continue to focus on the rapid completion of our ongoing studies.”
In September 2016, Biogen will open the global expanded access program (EAP) for eligible patients with infantile-onset SMA (consistent with Type 1). In Canada, the EAP program is being initiated at existing nusinersen clinical trial sites where there is a path supporting long-term availability of nusinersen, namely at BC Children’s Hospital, the Hospital for Sick Children, Montreal Children’s Hospital, and Children’s Hospital – London Health Sciences Centre. Upon initiation of the EAP along with the appropriate local approvals, enrolment may start at individual sites after transitioning ENDEAR study participants to the open-label extension study.
If you are interested in obtaining more information on the EAP program, please contact the following lead investigators at either of the sites:
BC Children’s Hospital, Vancouver BC – Dr. Kathryn Selby
Hospital for Sick Children, Toronto ON – Dr. Jiri Vajsar
Montreal Children’s Hospital, Montreal QC – Dr. Maryam Oskoui
Children’s Hospital – London Health Sciences Centre, London ON – Dr. Craig Campbell
For more information on the specific study please go to