Elivaldogene Autotemcel

Form: Intravenous infusion

Strength: A personalized gene therapy designed to halt or slow ALD progression, dosed based on weight, administered as a single infusion in specialized treatment centers

Reference Brands: Skysona(US & EU)

Category: Cell & Gene Therapies

Elivaldogene autotemcel (Skysona) is regulated in the US and EU, supported by dossiers demonstrating safety, efficacy, and manufacturing quality in compliance with GMP and MDR standards. In the US, FDA approval involved extensive clinical validation, safety assessments, and comparability data. In the EU, CE marking certifies conformity with MDR requirements, with ongoing approval processes in some member states. These gene therapies undergo validation, stability testing, and audits, supported by comprehensive documentation including safety profiles, clinical trial data, and manufacturing practices. For licensing procedures, approved dossiers, and the latest regulatory updates, visit Pharmatradz. Ensuring regulatory compliance guarantees access to innovative treatments supporting early intervention in cerebral ALD worldwide.