Biosimilars: Technical and Regulatory Challenges Featured Report

15-4-FR-Coverby Cheryl Scott, Senior Technical Editor, BioProcess International

At least one innovator company has embraced the concept of biosimilars wholeheartedly, calling it “our next chapter in healthcare” in a 2016 report. That publication cites product characterization, preclinical studies, nomenclature, reimbursement, and regulatory pathways as the primary challenges facing companies in biosimilar development. (Note: See Amgen’s 2017 biosimilars report here.) For our own featured report on biosimilars this month, we asked the contributors about those topics. The answers below come from Bruno Speder (head of clinical regulatory affairs at SGS) and Mario DiPaola (senior scientific director at Charles River Laboratories).

Product Characterization: What is the most challenging aspect of biosimilar characterization? How are companies obtaining originator samples to compare against?

SPEDER: One major challenge in developing biosimilars is to obtain sufficient originator product for characterization testing in both the preclinical and clinical development phases. The supply of originator product is very closely monitored by the manufacturers of those products because they seek to slow down the development of biosimilars. So this can be a difficult task. Originator product samples usually are obtained through specialized distributors.

Preclinical Studies: Can nonclinical/animal studies aid in supporting extrapolation of indications? Continue reading “Biosimilars: Technical and Regulatory Challenges Featured Report”

How the commercialisation of gene and cell therapies can revolutionise modern medicine

‘If one or two CAR-T therapies get to market it will provide impetus and momentum behind the industry’ – Dr Akshay Peer, Vice President of Sales and Account Management at TrakCel discusses the revolutionary steps that cell and gene therapies are making through their increased commercialisation.

In a field that is constantly seeing developments, Dr Peer outlines CRISPR and CAR-T therapies as forms of gene editing that can dramatically change the face of gene therapies and their use within modern medicine. He believes that as the commercialisation of these treatments increases, the regularity of their use will as well.

This is due to the surge in ‘positive public opinion’ that will arise from the successful implementation of the therapies. Dr Peer identifies the positivity that already exists around the new therapies, with ‘everyone looking at when these therapies will come to market and how much they will cost’. Although not going into detail about the financial outcome, he outlines his hope that when the public can see the lives of adults and children enhanced by using these therapies, any uncertain opinions will change to ones of optimism and confidence.

Peer acknowledges the lack of information that can exist around the new therapies due to an unfamiliarity in their usage; ‘People can sometimes get ahead of themselves and not understand completely what we’re trying to do here in this industry’. However, he is sure that this will change once there is evidence of patients who are successfully treated.

There is clearly excitement developing around the use of gene therapies, outlined by Dr Peer.

Watch the full interview, filmed at Cell Therapy Manufacturing & Gene Therapy Congress, with Dr Peer above or here.

Extractables and Leachables: Standardizing Approaches to Manage the Risk [eBook]

15-3-March-eBookThe implementation, maturation, and benefits of single-use technologies in biopharmaceutical development and manufacturing are well documented and understood. As analytical methods and testing services also rapidly improve, it is clear that management of risk associated with extractables and leachables also must evolve. Standardization is universally accepted as a goal; how to define, implement, and educate the industry is where debate resides.

The container–closure segment has had more experience dealing with leachables and extractables than those implementing single-use process components do — they’ve just been doing it longer. BPI takes a closer look at relevant guidance and regulations and examines how groups such as BPSA, ELSIE, ISO, and USP are helping users make sense of it all.

This eBook reviews the industry’s approach to leachables and extractables to address an underlying question: How close are we to making standardization a reality?

Read an excerpt from the eBook below:

Continue reading “Extractables and Leachables: Standardizing Approaches to Manage the Risk [eBook]”

Efficient Integration of Single-Use Equipment During Capacity Expansion Projects

By Nick Hutchinson

More than ever before, biopharmaceutical companies are able to establish their own in-house biomanufacturing capabilities. The adoption of single-use technology has reduced the need for expensive utilities systems and large manufacturing footprints. The inherent flexibility of this technology is allowing firms to connect steps in the production process with relative ease and without the need for fixed stainless steel pipework. Upfront capital costs have diminished and although operating costs remain, they are incurred only when the success of a drug candidate or licensed product warrants further production. Thus, single-use technologies provide a means to mitigate the risk of wasting large capital expenditures in the event a molecule is unsuccessful in the clinic or on the market.

Good engineering practices are key

Single-use technology is available for nearly every step in a biopharmaceutical manufacturing process below a certain scale of production. Biologics such as monoclonal antibodies and viral vaccines can be produced using processes in which the entire product, media and buffer flow-paths are disposable. However, companies attempting to install or expand new biomanufacturing capacity should be mindful that they should follow good engineering practices to maximize the probability of success. Despite the ease with which firms can install single-use capacity, relative to traditional stainless steel projects, this can nevertheless lead to an insufficient consideration of how firms should integrate single-use equipment with other steps in the process chain. The overlooking of proper integration can lead to incorrect equipment sizing, poor equipment design or an incomplete solution being developed. This can result in process failures, delays and the need to perform costly engineering rework.

Continue reading “Efficient Integration of Single-Use Equipment During Capacity Expansion Projects”

Breakthrough Technologies in Cell and Viral Therapies

BPI West: Day 1 Recap

“The future disruption of therapeutic modalities is closer than we think.” – Uwe Gottschalk

In Track 3, Late Stage Process Development & Commercial Launch Preparation, for a standing room only crowd, the featured presentation was given by Uwe Gottschalk, Chief Scientific Officer at Lonza. This track was developed to help attendees learn best practices to apply new technologies used by industry leaders to mitigate risk, improve product quality and ensure regulatory compliance for promising biologics. This exciting presentation included discussions around; T-Cell based cancer treatments, an overview of allo vs. auto, the challenges in scaling of products, the need for automation, disruptive technology and much more.

Continue reading “Breakthrough Technologies in Cell and Viral Therapies”

The Recent Growth of Biotech Clusters Around the Globe

David Brindley, Senior Research Fellow at University of Oxford Department of Pediatrics comes from a very interesting background that covers both academia and industry. He joined us during Biotech Week Boston to discuss the relationship between academia and industry, what he identifies as the key regulatory challenges in both the US & Europe, and then finally he explores the recent growth of biotech clusters around the globe.

By design, there are a number of initiatives around that globe to create biotech clusters. “The two greatest biotech clusters in the world are undoubtedly the 128 corridor around Boston and Silicon Valley on the West Coast” Brindley says. However, he does mention that these clusters were development completely by accident, and not by design. Moving forward, as the growing of biotech cluster initiatives progress, these regional hubs need to focus/specialize on a very deep niche. If you are looking to set up a new cluster, Brindley says, “you should worry less about the platform technology it will develop,  is it monoclonal or is it small molecule. And [instead], focus more on ‘what is the bioprocessing problem that we are going to address here? Is this going to be a center of manufacturing?’ because I think even saying we are going to be a center of drug development is too broad.” This train of thought shows that collaboration is necessary for large-scale success of the industry.

Continue reading “The Recent Growth of Biotech Clusters Around the Globe”

Affordable Biologic Downstream Purification with Single-Use Protein A Membrane

This article was originally published on www.DownstreamColumn.com by Brandy Sargent, Editor in Chief

At this year’s Biotech Week Boston there were many exciting talks on downstream purification and associated new technologies. In particular, there were several talks about optimizing the downstream purification process. One very interesting talk, given by Renaud Jacquemart, PhD Principal Scientist, Director Vaccines Process Sciences, was titled “Enabling Manufacturing Of Affordable Biologics Through The Use Of A Protein A Membrane
 In A Single-Use Purification Strategy ” and focused on the application of a fully single-use chromatography purification process in place of resins. This strategy envisions the use of a unique Protein A membrane for which Natrix recently signed collaboration agreements with Merck & Co. and Sanofi.

Creating a more affordable purification strategy

In his talk, Dr. Jacquemart begins by talking about the goal of creating a more affordable purification strategy and how the Natrix approach incorporates a holistic vision of the entire manufacturing process. To meaningfully decrease total cost and create the most efficient process, companies must significantly reduce the physical scale of manufacturing facilities and enable greater flexibility. This permits faster turnaround and accommodates a wider range of scales and products for any given time period. Achieving these goals requires a large increase in productivity and much-simplified single-use architecture for purification.

Continue reading “Affordable Biologic Downstream Purification with Single-Use Protein A Membrane”