In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging results from the late-stage CHERISH clinical trial to test the experimental drug nusinersen (brand name Spinraza) in children with type 2 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label trial called SHINE, in which all children will be treated with the drug.
Learn more about today’s news, including a Q&A provided by Biogen and Ionis, at Encouraging Results in Nusinersen Trial for Type 2 SMA. In addition, read a message the two companies have issued to the SMA community:
Dear Members of the SMA Community,
Today we take another important step to advance the first potential treatment option for Spinal Muscular Atrophy (SMA). We are happy to continue to provide exciting news about the Spinraza (nusinersen) program by sharing positive results from a pre-specified interim analysis of the CHERISH study in later-onset (consistent with Type 2) SMA. The analysis found that children receiving Spinraza experienced a statistically significant improvement in motor function compared to those who did not receive treatment. Spinraza demonstrated a favorable safety profile in the trial. With the positive interim analysis, the CHERISH study will be stopped and participants will be able to transition into the SHINE open-label extension study to receive SPINRAZA.
We conducted detailed scientific analyses using all the available data to identify the earliest point in the CHERISH study when an interim analysis could be conducted to provide us with the well-controlled data needed to help assess the safety and efficacy of Spinraza. We believe this analysis provides further evidence of Spinraza’s potential impact on SMA. We will make regulators around the globe aware of these data and work with them to identify the most efficient path toward achieving our goal of bringing Spinraza to the SMA community as quickly as possible.As we have previously communicated, Spinraza has received Accelerated Assessment status from the EMA and the FDA plans to act early on our NDA under expedited review. There are still a number of steps to go, but Biogen is preparing for the potential launch of Spinraza in the U.S. possibly as early as the end of 2016 or the first quarter of 2017.
Biogen recently initiated a global expanded access program (EAP) in infantile-onset SMA in October. Given the complexities and operational aspects of opening an EAP in SMA, the initial phase in infantile-onset has taken longer than expected to open. In addition, we recognize the anticipated expedited regulatory timelines in the U.S. may not allow for the expansion of the EAP for Type 2 patients in the U.S. prior to a potential approval. However, we will continue to explore the ways in which we could broaden the EAP to include patients with Type 2 SMA in geographies where the time to regulatory approval is anticipated to be longer. Regulatory approval remains our number one priority as we continue to believe it is the best way to provide broad and sustainable access to the SMA community.
Every day, we are spurred on by the stories we hear from the SMA community and are very grateful to all the families participating in our clinical studies as well as the physicians who work tirelessly to care for them day in and day out. We will continue our relentless pursuit of a treatment option for the community and continue to share information and program updates to the community when possible. We would like to express our deepest appreciation to all involved in this important journey.
Biogen and Ionis Pharmaceuticals
