Gene Therapy CMC Guidance Released

The US Food and Drug Administration issued a final guidance last week regarding investigational new drug applications for human gene therapy. Although gene therapy is still in the developmental stages, there is much hope for gene-based treatment and products. Because the therapy involves replacing a patient's faulty genetic material with normal genetic material,there is concern about safety in making sure replacement cells continue to function as they should. The guidance, therefore, focuses on the type of information that scientists need to consider and include in their documentation when studying these drugs and submitting them for FDA approval.

Based on a draft guidance that was issued in November 2004 (69 FR 64958), the final document, Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), provides information on what to include in an original IND. The document also applies to combination products that contain a human gene therapy biological product in combination with a drug or device as part of the final product.

The full guidance is available for review and comment here.