Preparing for a Growing Biologics Market

BioPharm International spoke with Cindy Reiss-Clark, vice president, general manager, Biologics at West Pharmaceutical Services Inc., about the growing biologics market, specifically gene therapies, and how West is helping their clients prepare for these new trends.

BioPharm: How has the biologics market grown during the past five years? 

Reiss-Clark: The biologics market has experienced double digit growth over the past five years. We see value outpacing volume. Volume is not growing at the same pace as the value of the market due to a focus on rare and ultra-rare diseases. The way West tracks its molecules in PharmaCircle, we see cell and gene therapy treatments making up ~25% of the biologics molecule pipeline. Other than CNS [central nervous system] diseases like Alzheimer or Parkinson’s, there are treatments for most of the big patient population diseases, and to show significant improvement in patient outcome is difficult. Many molecules are now targeting much smaller patient populations.

BioPharm: What are the benefits of gene therapy? How is the gene therapy market expected to grow in the next five years?  

Reiss-Clark: Gene therapy has the promise with as little as one dose to eliminate the underlying cause of disease. This can dramatically change the lives of patients and their treatments.

There are now gene therapies approved and on the market, so we expect this market to continue to grow. At the recent PDA Cell and Gene Conference in Long Beach, CA Sebastian B. Teitz, PhD presented a slide quoting Scott Gottlieb expects the FDA to approve 40 gene therapies by 2022 (FrontLine Genomics, Jun. 2018). Thomas Leitch from Bluebird Bio, at the same conference, stated 39 by 2022. There are over 400 products in clinical trials, and 25% of biologics in clinical phases are a cell or gene therapy.

BioPharm: What role do viral vectors play in the manufacture of gene therapies?  

Reiss-Clark: Without vectors, there is no gene therapy. Vectors are the delivery mechanism to get genetic material (gene therapy) into the cell. There has been a push to try nonviral vectors, but they just don’t have the gene delivery horsepower that viral vectors have.

BioPharm: What investments has West made in the biologics market? Have there been any specifically for gene therapy?   

Reiss-Clark: West has made significant investments into the biologics market. In partnership with Daikyo Seiko, Ltd., we brought FluroTec barrier film to market in the early 90s specifically to address these sensitive molecules. Over the years, we continue to bring specific materials and quality standards like NovaPure components to address the ever-increasing pressure on particulates.

West recognized the promise of gene therapies several years ago and has been targeting companies in this space with innovative packaging solutions and services, specifically leveraging Daikyo Crystal Zenith (CZ) technology for its physical properties, break resistance, and low temperature compatibility, which is required in this space.

BioPharm: What services/capabilities does West offer when it comes to gene therapies? 

Reiss-Clark: We are always reviewing the ever-changing needs of this market. Our current offerings are in our core areas of containment with strategies to expand in other areas.

Currently, we believe our CZ offering is a real workhorse in this area for its physical properties, break resistance, low temperature compatibility, and good particle profile.   CZ in addition with our NovaPure stoppers with FluroTec film to protect our client’s valuable product from extractables and reducing any risk of the viral vectors adsorbing to the stopper. is our recommended containment system.   In addition to the containment systems itself, we also have a wide option of integrated solutions, like testing services such as extractable and leachable and container closure integrity (CCI) testing to assist customers in getting to market.  

Speed to market is critical, as many customers have been granted accelerated approval status, breakthrough status, or fast track status by the FDA or other regulatory bodies. By providing small quantities with quick turnaround (Ready Pack off the shelf vial containment system), we have been able to get customers product they need in a timely manner. Our service offerings have enabled customers to focus on their drug product (what they do best) while West handles what we know best, containment systems and the necessary requirements to get them successfully to market. 

Current gene therapies on the market are mostly for rare disease, and companies are shifting to diseases like hemophilia with larger patient populations. These drugs can have dramatic consequence for patients suffering with such diseases. We will continue to review our product and service offering to ensure we are focusing on the right solutions for this important area of growth.