Biologics

Guide on Gene Therapies for Neurodegenerative Diseases

FDA has released a final guidance entitled “Human Gene Therapy for Neurodegenerative Diseases.” The document focuses on considerations for product development, preclinical testing, and clinical trial design.

The guidance says that general chemistry, manufacturing and controls for product manufacturing, testing and release of gene therapy products should address challenges associated with the route of administration, volume of product that can be administered, the delivery device, and the study population size.

“Typically, critical quality attributes (CQAs) of an investigational drug product,” it says, “are evaluated during each phase of clinical development, and characterization data from multiple drug product lots are correlated to clinical outcomes. Early-phase clinical studies of neurodegenerative diseases involving small study populations, in addition to focusing on safety assessments, may also provide early evidence on effectiveness. In cases where early-phase clinical studies are used to provide evidence of effectiveness to support a marketing application, the product’s CQAs and manufacturing critical process parameters (CPPs) should be thoroughly evaluated and appropriate controls implemented during the early clinical development phase.”

Additionally, the agency says innovative manufacturing strategies such as the production of multiple small lots versus a single large product lot may be considered to increase manufacturing process experience and product knowledge. Because gene therapy products may have quality attributes with higher variability than typical products, FDA recommends sponsors consider additional product characterization studies to establish acceptance limits for the CQAs. “For licensure, it is important to demonstrate process control to ensure a consistent product with predefined CQAs for product strength (e.g., vector genomes/mL), potency, identity, and purity,” it says.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome