Biogen Inc. — Spinraza FDA Review (NDA209531, ORIG 1, 2016)
Biogen Inc. · 2016 · FDA Review (openFDA) · company profile →
Report summary
The FDA review of Spinraza (nusinersen) for Spinal Muscular Atrophy (SMA) details both its efficacy and safety. Clinical trials, particularly the sham-controlled study CS3B, demonstrated significant improvements in motor milestones, CHOP INTEND scores, and survival rates in infantile-onset SMA patients. Secondary endpoints also showed consistent benefits. However, the safety profile revealed several concerns. Common adverse events included respiratory infections and constipation. More serious issues noted were thrombocytopenia, renal toxicity (proteinuria, low serum bicarbonate), hyponatremia, decreased growth, and potential hepatotoxicity (elevated liver enzymes). Other findings included QTc prolongation, vasculitis, and neurologic toxicity. The report recommends careful monitoring for these adverse effects and proposes specific labeling and post-marketing surveillance strategies. The drug was found to have no abuse potential.
Key findings — cited to the page
| ECG shift to abnormal, clinically significant post-baseline | 8 % | Study CS3B · p.18 |
| Treatment-emergent elevated urine protein (trace level or higher) in sham control | 20 % | Study CS3B · p.19 |
| Thrombocytopenia incidence (< 100 x 10^9/L) | 2 % | all nusinersen subjects · p.16 |
| Adverse event (bradycardia) in sham control | 10 % | · p.17 |
| ADA-positive results | 1-5 % | · p.18 |
| Treatment-emergent elevated urine protein (trace level or higher) | 33 % | Study CS3B · p.19 |
| PDUFA Goal Date | May 23, 2017 | Spinraza (nusinersen) · p.2 |
| Thrombocytopenia incidence | 11 % | Study CS3B · p.16 |
| Shift from baseline to high aPTT | 19 % | · p.17 |
| Adverse event (bradycardia) | 6 % | · p.17 |
| QTcf above 500 msec and increase of > 60 msec from baseline | 5 % | Study CS3B · p.18 |
| Treatment-emergent elevated urine protein (trace level or higher) in later onset SMA | 69 % | Pool E · p.19 |
Source: Biogen Inc. ↗ · License: US Government public domain. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →