Biologics

Gene Therapy Guide on NonClinical Biodistribution

FDA has released an International Council for Harmonization final guidance entitled “S12 Nonclinical Biodistribution (BD) Considerations for Gene Therapy Products.” It provides harmonized recommendations for the overall design of nonclinical BD assessments. Considerations for interpretation and application of the BD data to support a nonclinical development program and the design of clinical trials are also contained in the document.

According to the guidance, BD is the in vivo distribution, persistence, and clearance of a gene therapy product at the site of administration and in target and nontarget tissues, including biofluids (e.g., blood, cerebrospinal fluid, vitreous fluid). “Nonclinical BD assessment entails the use of analytical methods to detect the GT product and transferred genetic material in collected samples and can include methods to detect the expression product of the transferred genetic material,” it says.

The guidance says that BD data should be available when evaluating and interpreting the nonclinical pharmacology and toxicology findings. “Nonclinical BD data can also inform design aspects of a first-in- human clinical trial (see section VI (6)),” it says. “Nonclinical studies for BD assessment can be conducted as stand-alone BD studies or in conjunction with nonclinical pharmacology and toxicology studies. “

Additionally, the document recommends that nonclinical BD studies should be performed in a biologically relevant animal species or model following gene therapy administration that is representative of the intended clinical product. “It is important that the route of administration (ROA) reflect the intended clinical ROA to the extent possible and that the dose levels studied provide sufficient characterization of the BD profile,” it adds. “It is important to verify the data quality, integrity, and reliability of the BD evaluation.”

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