Pharmaceutical Consulting and Project Management: Preclinical - CMC - Regulatory

The field of nucleotide-based therapies continues to make progress. Several products have already gained FDA approval including Macugen TM (Pegatinib), a PEGylated aptamer for wet age-related macular degeneration (AMD). Many more are in late stage clinical trials. Regulatory requirements may differ for these different types of drugs compared to what is required for an NCE. The main challenge is targeting the delivery which may require the use of nanoparticles or lipid formulations, or attaching a carrier molecule such as a virus, antibody or peptide. When assessing the safety of a nucleotide-based therapy, both the delivery vehicle and the nucleotide product itself have to be evaluated. More...

It seems that interest is growing for starting Phase 1 trials in Australia due in part to the comparative ease of regulatory submission, the scientific confidence in the results and the lower costs. There is quite a bit of regulatory history in Australia that might contribute to a combination of adherence to good clinical practices and favorable regulatory procedures. More...