Earlier initiation of treatment with Spinraza (nusinersen) may improve outcomes in children with spinal muscular atrophy.- Biogen.

Biogen presented new data demonstrating that earlier initiation of treatment with Spinraza (nusinersen) may improve motor function outcomes in infants and children with spinal muscular atrophy (SMA). Results continued to reinforce the favorable efficacy and safety profile of Spinraza. The data were shared at the 22nd International Annual Congress of the World Muscle Society in Saint Malo, France (October 3-7, 2017).

A new analysis from the Phase III ENDEAR study showed infants with SMA who initiated treatment earlier in the disease (shorter disease duration) demonstrated greater benefit and improvement in motor function outcomes. As measured by the Hammersmith Infant Neurological Examination (HINE), significant differences in motor milestone responders were observed between infants treated with Spinraza compared to untreated infants with disease duration less than or equal to 12 weeks (75% vs. 0%; P<.0001) and those with disease duration greater than 12 weeks (32% vs. 0%; P=.0026). There was also a significant benefit in event-free survival in infants treated with Spinraza with disease duration less than or equal to 12 weeks (P=.0004). Interim analyses were also presented from the Phase II EMBRACE study which was designed to assess the efficacy and safety of Spinraza in individuals with infantile- and later-onset SMA who were ineligible for the two earlier pivotal studies. The EMBRACE interim analysis showed a larger proportion of infants and children treated with Spinraza were HINE motor milestone responders compared to those who were untreated. Results from the interim analysis also supported the dosing regimen of four loading doses in the first two months, followed by the administration of Spinraza every four months thereafter, for individuals with infantile- and later-onset SMA.

In the ENDEAR and EMBRACE studies Spinraza demonstrated a favorable benefit-risk profile. Safety data involving the intrathecal administration of Spinraza showed the incidence and nature of the most common lumbar puncture-related adverse events in the clinical studies were similar in children with later-onset SMA with or without scoliosis.