Veristat Supported Eight Regulatory Approvals in 2022, the Majority for Therapies Designed to Treat Rare Diseases

Veristat, a scientific-minded global clinical research organization (CRO), announced today that the Company supported clients in preparing marketing applications for eight therapies that received regulatory approval in 2022. The approvals from the US Food and Drug Administration (FDA) included one Biologics License Application (BLA), five New Drug Applications (NDAs), and one Supplemental New DrugApplication (sNDA). One Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) was approved, and collectively most of the approvals were for therapies targeting a rare disease. Veristat’s success in preparing marketing applications that receive approval can be attributed to the ability of our integrated teams to navigate the complex regulatory submission process with the FDA and regulators around the world.

“At Veristat, we believe championing better health not only equates to finding cures for diseases but also improving patients’ abilities to more ably live with their disease,” stated Patrick Flanagan, Chief Executive Officer of Veristat. “A key measure of our success is how effectively we advance the numerous possibilities for novel innovation to improve the quality and duration of human life. While it’s important to recognize our progress in advancing treatments through the development and approval process, there is more work to do, particularly in the area of rare diseases.”

According to the National Institutes of Health (NIH), there are approximately 7,000 rare diseases and only 5% of known rare diseases have one or more approved treatments. With February Rare Disease month, Veristat teams reaffirm their commitment to advancing rare disease research across several modalities that span gene therapies, cell therapies, and tissue engineering— including the first gene therapy approved in the Western world.

In the last three years, Veristat teams have:

• Conducted 190+ rare disease projects for over 110 sponsors working on rare disease treatments
• Prepared 30+ INDs and CTAs for rare disease therapies
• Supported 55 Marketing Applications/Authorizations, of which 40% were for rare disease therapies
• Celebrated the success of clients receiving more than 25 regulatory approvals for marketing applications prepared by Veristat teams, 75% of which were for rare disease therapies.

From regulatory pathway selection to patient recruitment and site access difficulties to the intricacies of data collection, a rare disease therapy requires an extraordinary amount of coordination and scientific understanding. Effective utilization of natural history studies and decentralized clinical trial deployment can be valuable.

“Rare disease research presents many challenges for trial design and regulatory strategy,” stated John Balser, Ph.D., Veristat Founder, President, and Chief Statistical Officer. “Small population sizes, uncertainty in clinical endpoint requirements, and an often limited understanding of the physiological basis of the disease can make for a difficult path forward. Over 30% of Veristat’s work is in rare diseases. The knowledge we have amassed has been game-changing for sponsors. Our creative approaches have helped them overcome daunting evidentiary and regulatory hurdles to gain swift and successful therapy approval.”

About Veristat

Veristat, a scientific-minded global clinical research organization (CRO), enables sponsors to solve the unique and complex challenges associated with accelerating therapies through clinical development to regulatory approval and commercialization. With more than 28 years of experience in clinical trial planning and execution, Veristat is equipped to support any development program.

Veristat's focus on novel drug development has led to success when handling the unknowns that arise across complicated therapeutic areas, such as rare/ultra-rare disease, advanced therapies, oncology, and infectious disease trials. Every day, we apply this knowledge base to solve any program’s clinical, regulatory, statistical, data, or operational challenges, from the simplest to the most complex. Veristat has assembled an extraordinary team of experts worldwide who have mastered therapeutic development intricacies, enabling sponsors to succeed in extending and saving patients’ lives.

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Contact information

Lauren L. Brennan, Vice President of Marketing, Veristat
media@veristat.com