Onasemnogene Abeparvovec

Form: Injectable gene therapy

Strength: Single-dose IV infusion providing 6 x 10^13 vector genomes (vg) per patient

Reference Brands: Zolgensma(US & EU)

Category: Cell & Gene Therapies

Onasemnogene abeparvovec (Zolgensma) is regulated in both the US and EU, supported by dossiers demonstrating safety, efficacy, and manufacturing quality following GMP standards. In the US, FDA approval involved extensive clinical trials, safety evaluations, and GMP compliance. In the EU, CE marking certifies conformity with MDR standards for pediatric spinal muscular atrophy (SMA) treatment. These therapies undergo validation, stability testing, and audits, with detailed documentation including clinical trial data, safety profiles, and manufacturing practices. For licensing procedures, approved dossiers, and latest regulatory updates, visit Pharmatradz. Ensuring compliance grants access to innovative, life-changing gene therapy for SMA worldwide, supporting early intervention and improved long-term outcomes.