Tuesday, March 25, 2014

BDP Week: Lifecycle Management of Processes for the Production of Biopharmaceuticals for the Treatment of Rare Diseases

The IBC Life Science Biopharmaceutical Development and Production Week in San Diego conference kicked-off the week on Monday March 24th with Joanne Beck, Vice President of Process Development at Shire, featured as a Keynote Speaker. The title of her talk was, Lifecycle Management of Processes for the Production of Biopharmaceuticals for the Treatment of Rare Diseases. Biopharmaceutical production for rare diseases meant greater challenges in the area of life cycle management of those processes because it involved orphan drug designation by the FDA with which hand-in-hand came accelerated development. A critical approach at Shire in life cycle management is to balance risk and product quality and availability for each of the one in 10
patients affected by one of the 7000 rare diseases of whom 50% are pediatric. The case studies presented by Beck included the production of biopharmaceuticals for the treatment of dystrophic epidermolysis bullosa, or DEB, which is a genetic connective tissue disorder, and two lysosomal storage disorders, one being metachromatic leukodystrophy, or MLD, and the other being Hunter syndrome. In each case, the biopharmaceutical being manufactured was for use in a protein replacement therapy, or in the latter two cases, more specifically an enzyme replacement therapy.

The lifecycle management of the manufacturing process faced quality challenges such as purity and complex structural integrity of the protein necessary for proper function. The challenges were overcome by optimization of cell culture production to improve yield which involved parallel paths of attack such as pursuing alternative host cell lines to not only address yield but also the impurity profile, development of improved analytical methods such as for enzyme specific activity measurement, and fast-tracking formulation. In the case of MLD, an additional set of challenging issues was managing changes in manufacturing such as new unit operations, a new facility, and a new manufacturer, which in part involved bridging studies for drug product comparability, and new supply chain tracking protocol. In the case of DEB, unique challenges arose from the technology being acquired from a virtual company which necessitated that a first step be the aggregation of all existing data for complete and thorough analysis and review by Shire in order to avoid going down the path of reinventing the wheel. And lastly, in the case of Hunters, life cycle management addressed challenges in a retrospective quality by design fashion by “looking at old data through new lenses” to develop some fundamental understanding of critical parameters required for catalytic activity.

In summary, Beck cited success as having to do with development and implementation of science and risk management tools, identifying and building core capabilities, focusing on critical steps at every stage, and streamlining work-flows for design and planning. The conference continues through Thursday March 27th with talks and workshops in four to five daily session topic tracks at the Hilton Bayfront Hotel.

Today's post comes from Gregory T Mrachko a Protein Chemist and Enzymologist. He specializes in protein purification, enzymology, high throughput assay/screen conceptualization/development/implementation, analytical biochemistry, biocatalysis, liquid column chromatography, and protein analytics. He is a guest blogger at this year's Biopharmaceutical Development and Production Week and can be reached at gmrachko@msn.com.

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