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Gene therapies are developed by modifying a person’s genes and are often targeted to treat complex and chronic diseases. Due to advancements in science and research, gene therapy is positioned to transform treatment options for patients who are living with these incurable diseases. There is a growing pipeline of gene therapies globally, with 352 global trials underway at the end of 2019, up 12% from 2017.1.
As advancements in gene therapies continue to grow, West is committed to providing packaging solutions for these complex treatments. Learn more about a biotechnology company, with a recently approved gene therapy, that partnered with West to select a packaging solution that would allow them to meet their needs for storage at low temperatures and still get to market quickly.
The Challenge
Bringing the first gene therapy for pediatric Spinal Muscular Atrophy to market in a leak and break resistant container which maintains sterility over product shelf life, through freeze and defrost cycles. Container Closure Integrity (CCI) must be proven and consistent for storage at -80°C, and the solution must be ready to scale-up quickly due to orphan designation and Fast Track status of the program.;
The Considerations
When planning gene therapy drug development, it is critical to ensure known challenges are mitigated; therefore, a chosen packaging system needs to be proven, reliable and able to be scaled-up, at short notice. The supply chain for gene therapies requires the drug to be frozen, then stored at -80°C before being thawed for injection into the patient. It is critical that packaging components used in this complex supply chain are compatible with both the drug and specialized conditions required for storage and distribution. Having secured Fast Track designation from the FDA, the customer was able to bring its gene therapy to market quickly, allowing patients with the highest need to receive treatment in record time. The packaging system selected helped to facilitate the speed with which the drug moved from development to commercialization. The chosen system provided a range of container sizes with quality components, in ready-to-use format, proven to work as a system and available in small quantities.
The Solution
West’s Ready Pack™ system was selected, which includes a Daikyo Crystal Zenith® vial, NovaPure® stopper and Flip-Off® CCS seal. These high-quality components combine to create a proven system which ensures CCI over theproduct shelf life and low temperatures. In 2019 the FDA approved the first gene therapy treatment for pediatric Spinal Muscular Atrophy using West’s container system.
Customers can purchase the small quantity Ready Pack system directly from West’s online store. Buying this offering online allows for quick delivery and enables customers to meet the short timelines often associated with orphan drug designation and Fast Track approval. Daikyo Crystal Zenith vials and associated West components were the right choice for this customer, as vial sizes from 2-5mL supported expedited scale-up activities from lab to the patient. The Ready Pack system offers all these benefits without the unwanted cost of excess stock and is the perfect choice for bringing a gene therapy to market.
Ready Pack™, NovaPure® and Flip-Off® are trademarks and registered trademarks of West Pharmaceutical Services, Inc., in the United States and other jurisdictions.
Crystal Zenith® is a registered trademark of Daikyo Seiko, Ltd.
Crystal Zenith® technology is licensed from Daikyo Seiko, Ltd.
Research in the area of cell and gene therapies started almost 40 years ago, however recently there has been a been a surge of activity including landmark regulatory approvals for therapies treating chronic and rare diseases. In 2017 and 2018 the FDA approved the first two CART-T cell therapies for cancer treatment and the first two gene therapies for rare diseases. All four of these cell and gene therapies were approved under the FDA Accelerated Approval Program, aimed at expediting approvals for life saving drug therapies. These regulatory programs have the potential to shorten drug development as much as 1-3 years. Currently, there are more than 1,000 regenerative medicine clinical trials underway globally<sup>1</sup>, and the market is expected to grow rapidly over the next 10+ years.
It is an exciting time to be in the Cell and Gene therapy industry. The astounding growth and development we have witnessed in the cell & gene therapy industry over the last decade is bringing life-changing therapies to the patients who have been so anxiously awaiting them. <br /><br />At the same time, as with any new drug class, challenges that may not have been considered during the drug development phase may suddenly show up and stand in the way of overcoming that last hurdle: Packaging and delivering your precious new molecule to the patient.
Competition is increasing in the biopharmaceutical pipeline due to a growing number of new entrants, more players are bringing their drug all the way to market and many companies are utilizing accelerated development pathways, making it an even faster race to market. If you are like many other newly formed biotech companies, you’re doing your best to manage many activities critical to your success with limited resources, all while trying to accelerate your path to market.
Biologics is a rapidly growing market, with a robust R&D pipeline bringing innovative therapies to treat patients. Today, there are 4,000 more active biologic programs in the pipeline compared to 2015, with biologics development programs growing 2.5 times faster than small molecules. New entrants into the biologics market are driving this rapid growth. As of 2018, emerging companies represented 84% of all early phase research.<sup>1</sup> Not only is this segment driving R&D growth, but they are having commercial success in bringing their drugs to market. In 2018, 65% of new drugs launched were patented by emerging companies.<sup>1</sup>
West is pleased to announce that we’ve recently expanded our Online Store offerings to include a 20mm NovaPure<sup>®</sup> Ready Pack™ lyophilization stopper, adaptiQ<sup>®</sup> vials from SCHOTT and Ready Pack™ Flip-Off<sup>®</sup> CCS (Clean, Certified, Sterilized) seals. West’s Online Store is a one-stop solution for access to small quantities of some of West’s highest quality products, in stock and available ready-to-use in convenient small packs. Depending on their product needs, shoppers can now purchase a complete vial containment system with the option of selecting either Daikyo Crystal Zenith<sup>®</sup> or adaptiQ glass vials.
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