Spinraza

Spinraza (nusinersen) is a drug indicated for the treatment of spinal muscular atrophy (SMA). Regulatory review documents indicate that in clinical trials, Spinraza demonstrated an improvement in motor milestone development in 40-41% of treated patients, compared to 0% in control groups. Specific motor milestone achievements included full head control in 18% of patients, independent sitting in 10%, and standing in 2%. The drug also led to a 65% improvement of at least 4 points in the CHOP INTEND score, versus 4% in the control group. Mortality was observed in 15% of treated patients, compared to 32% in control groups.

Key safety findings included thrombocytopenia incidence of 11%, with 2% experiencing levels below 100 x 10^9/L. A shift from baseline to high aPTT occurred in 19% of patients. Treatment-emergent elevated urine protein (trace level or higher) was observed in 33% of patients, compared to 20% in sham control. Bradycardia was reported as an adverse event in 6% of patients, versus 10% in sham control. The drug received Orphan Drug Designation on April 18, 2011, and Fast Track designation on November 29, 2011. Rare Pediatric Disease Designation was granted on October 21, 2016.