Charles River Laboratories Expands Access to Pediatric PDX Collection Following FDA/EMA Regulations Mandating Pediatric Testing for New Oncology Drugs
Charles River Laboratories International, Inc. (NYSE: CRL) announced a new resource for advancing pediatric oncology research. The company is now providing access to ITCC-P4’s well-characterized collection of 400 annotated pediatric cancer models. This initiative aligns with the new regulatory requirements set by the U.S. Food and Drug Administration’s (FDA) Research to Accelerate Cures and Equity (RACE) for Children Act and European Medicines Agency (EMA) regulations, making pediatric testing a prerequisite for oncology drug development.
The Food and Drug Administration Reauthorization Act (FDARA) introduced a significant change in pediatric oncology research with the amendment of Section 505B of the Food, Drug, and Cosmetic Act (FD&C Act), specifically under Section 504. This amendment, effective for original applications submitted after August 18, 2020, requires pediatric investigations for certain targeted cancer drugs with new active ingredients. These investigations are based on the drugs’ molecular mechanism of action rather than clinical indications. In essence, if an original New Drug Application (NDA) or Biologics License Application (BLA) is submitted for a new active ingredient designed for treating adult cancer, and it targets a molecular mechanism that the FDA deems substantially relevant to pediatric cancer’s growth or progression, the application must include reports on the molecularly targeted pediatric cancer investigation. This requirement is mandatory unless the FDA chooses to waive or defer it. This change aims to expedite the development of effective therapies for children with cancer, providing an essential response to the unmet medical needs in the pediatric population. Unlike previous regulations that were based on clinical indications, this shift recognizes that pediatric and adult cancers may share the same molecular abnormalities, despite their differences in origin, biology, and natural history. Large-scale efforts in pediatric cancer genome sequencing have provided evidence that some genetic vulnerabilities in adult cancers also exist in pediatric cancers. This has paved the way for an enhanced focus on precision medicine in pediatric oncology.
The ITCC-P4 collection grants access to models tailored to the unique characteristics of pediatric cancer. These models allow researchers to delve into specific targets of interest, ultimately facilitating the translation of research into preclinical trials. Pediatric tumors differ significantly from adult tumors in terms of genomic drivers and phenotypes, necessitating specialized preclinical models. The ITCC-P4 collection is further supported by a wealth of data encompassing molecular phenotyping and pharmacological characterization. This comprehensive dataset is bolstered by the expertise of internationally recognized pediatricians, ensuring the utmost accuracy and relevance in pediatric oncology research.
Aidan Synnott, Corporate Vice President, Global Discovery Services at Charles River, emphasized the significance of this development. He stated, “Globally, 400,000 children and adolescents develop cancer each year, and approximately one in four cannot be cured with currently available therapies. The ITCC-P4 collection is comprehensive, relevant, and well-characterized, meaning we can better assess the safety and efficacy of new oncology treatments specifically for children. This will ultimately lead to new treatment options for a critically important patient population.”