Orphan Drug Development for Rare Diseases

With the advances in medical and biotechnology research, the pharmaceutical and biotechnology industries are able to address extremely rare and acute orphan diseases that in the past had no treatment.  Whether through cell and gene therapy, specialized vaccines, or biologics these orphan drugs represent the leading edge of medical science. 

By definition, Orphan Drugs are niche products targeted to a very specific and limited patient population.  Because of the complexity of the manufacturing process and the limited patient pool, these products tend to be of extreme value to the company producing them.  Therefore, the manufacturing system to produce these products is required to maximize product yield for very small batches.  AST’s GENiSYS and ASEPTiCell aseptic filling and closing systems have innovative capabilities that maximize product yield and product quality.

Although differing in productive capacity both the ASEPTiCell and GENiSYS aseptic filling and closing machine have an innovative dosing system that ensures that each container processed has the exact amount of drug within the container.  Typically wasted product at the beginning and end of the batch represented many vials of wasted materials.  However, with AST’s in-process fill weight verification system (IPC), measurement of the material dosed into each container can be accomplished in real-time and confirmed to be correct.