An early treatment with Spinraza improves motor function of patients with spinal muscular atrophy

This shows the new data from Biogen

New data from Phase 3 ENDEAR study demonstrated earlier initiation of treatment with SPINRAZA may improve motor function outcomes in infants with Spinal Muscular Atrophy (SMA)
Phase 2 EMBRACE interim analysis showed greater motor milestone achievement in infants and children treated with SPINRAZA, compared to those untreated, in patient populations not studied in the pivotal trials
Interim analysis of EMBRACE 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 infantile- and later-onset SMA

CAMBRIDGE, Mass.–Oct. 5, 2017– Biogen (NASDAQ: BIIB) 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 3 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). “These studies contribute to a growing body of evidence that SPINRAZA can make a meaningful difference in the lives of people with SMA regardless of their age or stage of the disease,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “Across studies, we continue to see evidence that earlier initiation of treatment with SPINRAZA can lead to improved clinical and functional outcomes.” Interim analyses were also presented from the Phase 2 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.

Mia

Mia

Mother of three childrens. One of the twins my eldest son had SMA Typ II -III. He passad away and spread his wings into the sun . He lived 3 years and seven months. Fly my sweet little honeybee🧚‍♂️🦋 fly my little Honeybee 🐝