Module 9 2021

Hazards of biotech products

• rHu Erythropoietin (various): variations in potency

• rHu Erythropoietin (Eprex): red cell aplasia

• rHu Factor VIII: suspected penicillium contamination

• PEG-rHu MGDF: thrombocytopenia

• Gene therapy: Gelsinger, X-linked SCID trials

• mAb TGN1412 : CD 28 agonist, phase 1 cytokine storm

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What happened on the journey?

Early on, it dawned on us that:

• Manufacturing technology was facile – Extraction with little or no processing – Collection and fractionation – Expansion/culture and modification

• High dependence on facility and raw materials

• Minimal analysis

• Inconsistency/quality attributes not detectable

• Manufacturing errors unchecked

• Biological activity/potency determined utility

The Organisation for Professionals in Regulatory Affairs

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