CAMBRIDGE, Mass. & CARLSBAD, Calif.–(BUSINESS WIRE)–Biogen (NASDAQ: BIIB) and Ionis (NASDAQ:IONS) today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Biogen has also applied for Priority Review which, if granted, would shorten the review period of nusinersen following the Agency’s acceptance of the NDA.
“Since announcing the positive results of the ENDEAR interim analysis in infantile-onset SMA last month, we have heard from many families expressing their excitement about nusinersen. Their stories continue to inspire us and they are in the forefront of our minds as we work to support the FDA’s review of nusinersen,” noted Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “We appreciate the FDA’s collaboration with us during the application process, and we look forward to continuing this productive dialogue, with the goal of rapidly bringing the first treatment for SMA to as many patients as possible.”
The Nusinersen Clinical Trial Program
The nusinersen clinical trial program is comprised of two controlled studies, ENDEAR and CHERISH. ENDEAR was designed as a thirteen-month study investigating nusinersen in 122 patients with infantile-onset SMA; the onset of signs and symptoms of SMA less than or equal to 6 months of age and age less than or equal to 7 months at screening. Based on the results of the pre-specified interim analysis, the ENDEAR study will be stopped; patients who elect to are currently being transitioned to the SHINE open-label study where they will all receive nusinersen. Results from the ENDEAR interim analysis will be presented at future medical congresses.
CHERISH is a fifteen-month study investigating nusinersen in 126 non-ambulatory patients with later-onset SMA, consistent with Type 2; onset of signs and symptoms greater than 6 months and age 2 to 12 years at screening. CHERISH was fully enrolled in May 2016 and remains ongoing.
Additionally, the SHINE open-label extension study, for patients who previously participated in ENDEAR and CHERISH, is open and is intended to evaluate the long-term safety and tolerability of nusinersen.
Two additional Phase 2 studies, EMBRACE and NURTURE, were designed to collect additional data on nusinersen. The EMBRACE study is designed to collect additional data on a small subset of patients with infantile or later-onset SMA who do not meet the age and other criteria of ENDEAR or CHERISH. Due to the evidence demonstrated in the infantile-onset SMA (most likely to develop Type 1) population, the sham arm of the EMBRACE study is being stopped and patients are being given the option to receive nusinersen through an open-label extension study. NURTURE is an open-label, ongoing study in pre-symptomatic infants who are less than or equal to 6 weeks of age at time of first dose to determine if treatment before symptoms begin would prevent or delay onset of SMA symptoms.