Etranacogene Dezaparvovec.

Form: Intravenous infusion

Strength: Single infusion product designed to produce sustained factor IX levels in hemophilia B patients, dosage individualized based on patient weight

Reference Brands: Hemgenix (US & EU)

Category: Cell & Gene Therapies

Etranacogene dezaparvovec (AMECVIT) is regulated in the EU and US, 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 regulations. These gene therapies undergo validation, stability testing, and audits, supported by comprehensive documentation including safety profiles, clinical trial results, and manufacturing practices. For licensing procedures, approved dossiers, and regulatory updates, visit Pharmatradz. Ensuring regulatory compliance guarantees access to innovative gene therapies supporting hemophilia B management worldwide, promoting effective treatment and improved patient outcomes.