Spinraza meets primary endpoint at interim analysis of Phase III study in later-onset SMA

Biogen is preparing for the potential launch of Spinraza in the US possibly as early as the end of 2016.

These results, along with our successful trial in infantile-onset SMA, reinforce the potential of SPINRAZA to benefit a broad range of SMA patients,” said Michael Ehlers, executive vice president, head of Research and Development at Biogen. “We will make regulators around the globe aware of this data and will continue working closely with them to bring Spinraza to families affected by SMA as quickly as possible.”

Biogen is preparing for the potential launch of Spinraza in the US possibly as early as the end of 2016 or the first quarter of 2017.

CHERISH is a 15-month study investigating Spinraza in 126 non-ambulatory patients with later-onset SMA (consistent with Type 2), including patients with the onset of signs and symptoms at greater than 6 months and an age of 2 to 12 years at screening.

Results from the primary endpoint of the pre-specified interim analysis demonstrated a difference of 5.9 points at 15 months between the treatment and sham-controlled study arms, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE). From baseline to 15 months of treatment, patients who received Spinraza achieved a mean improvement of 4.0 points in the HFMSE, whereas patients who were not on treatment declined by a mean of 1.9 points. The HFMSE is a reliable and validated tool specifically designed to assess motor function in children with SMA, and a change of three points or greater in the HFMSE has previously been identified as clinically meaningful. Data from the other endpoints analysed were consistently in favour of children who received treatment. Spinraza demonstrated a favourable safety profile. The majority of the adverse events were considered to be either related to SMA disease, common events in the general population, or events related to the lumbar puncture procedure. No patients discontinued the study. 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. Full study results will be presented at future medical congresses. “These data further validate the potential of Spinraza as a treatment for patients with SMA,” said B. Lynne Parshall, chief operating officer of Ionis Pharmaceuticals. “We are grateful to all the families and clinicians who have participated in all of the Spinraza studies. Without their commitment and support, this program would not have been able to progress so quickly.” FDA recently accepted the company’s New Drug Application (NDA) for Spinraza as a treatment for SMA and communicated they plan to act early on the NDA under an expedited review. In addition, the EMA recently validated Biogen’s Marketing Authorization Application (MAA) in the EU. The EMA’s Committee for Medicinal Products for Human Use (CHMP) granted Accelerated Assessment status and the FDA granted Priority Review to Spinraza. Biogen is initiating regulatory filings in other countries in the coming months. Biogen initiated a global expanded access program (EAP) in infantile-onset SMA earlier this year. The company will continue to explore where and when the EAP may be broadened to include patients with later-onset SMA (consistent with Type 2).