FDA releases draft guidance on next-generation sequencing (NGS) for genome editing safety assessment. Key implications for gene therapy manufacturers and IND submissions.

FDA updates SOPP 8212 on breakthrough therapy designation. Key changes in timelines, communication, and lifecycle management for manufacturers.

The FDA has issued a new draft guidance that updates the scientific principles for demonstrating biosimilarity. The document clarifies when strong analytical similarity data, supported by comparative human pharmacokinetic and immunogenicity assessments, may be sufficient to demonstrate biosimilarity without requiring a comparative clinical efficacy study. This updated approach can help manufacturers reduce development timelines and costs, while placing greater emphasis on robust analytical packages, sensitive PK study designs and a clear, risk-based justification aligned with FDA expectations.