Impact at a Glance
Regulatory Breakthrough
Enabled co-approval of Retevmo® and ODxTT for thyroid cancer in Japan.
Accelerated Timeline
Delivered a regulatory-ready, evidence-backed package in weeks instead of months.
Comprehensive Evidence Base
97 RET mutations rapidly mapped to clinical actionability and patient benefit.
Ongoing Support
Provided responsive scientific partnership throughout the PMDA review process.
The Customer Challenge
Loxo@Lilly set out to expand the regulatory label for Retevmo® (selpercatinib) in Japan, seeking approval for additional patient populations and broader mutation coverage using the Oncomine Dx Target Test (ODxTT). Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) presented a significant hurdle: Loxo was required to deliver comprehensive, reference-backed evidence for every RET mutation detected by ODxTT— demonstrating not just detection, but clinical actionability and predicted patient benefit. Loxo needed to respond within weeks, a timeline that far outpaced traditional internal data curation methods and risked derailing the label expansion effort.
Enabling Label Expansion with Literature-Derived Real World Evidence (RWE)
Genomenon partnered with Loxo@Lilly to unlock label expansion for Retevmo by delivering robust Real World Evidence (RWE) sourced from the global scientific literature. Genomenon provided a comprehensive dataset documenting the prevalence, activation status, and published clinical response data for each RET variant under consideration. By mining millions of full-text articles, Genomenon’s AI-powered platform and scientific team ensured that the expanded label reflected only mutations with clear, actionable benefit for patients.
Genomenon’s Strategic Difference for Loxo
AI-Powered, Human-Validated Curation: Genomenon’s proprietary AI scanned over 10 million scientific articles to identify every published RET mutation and all associated functional, clinical, and therapeutic evidence. Their 100+ expert scientists then applied rigorous review to ensure every variant was classified according to the latest guidelines and best practices—crucial for regulatory acceptance and label inclusion.
Comprehensive & Flexible Data: Loxo’s label expansion depended not just on variant detection, but on integrating multiple lines of evidence— activation status, clinical response, resistance mechanisms, and published patient outcomes. Genomenon’s methodology synthesized all of this into a single, regulatory-ready package, ensuring that only clinically actionable RET mutations were included in the expanded label.
Regulatory-Grade Transparency: Every piece of evidence was directly traceable to primary literature, allowing Loxo to respond confidently and transparently to PMDA inquiries—a necessity for successful label expansion and regulatory submission.
For Loxo, this meant:
- Rapid extraction, annotation, and interpretation of all 97 RET mutations detected by ODxTT, with evidence mapped to published literature and clinical guidelines.
- Delivery of a comprehensive “Variant Landscape” within weeks, formatted and reference-cited for immediate regulatory use.
- Ongoing regulatory partnership, providing on-demand scientific support and rapid response to PMDA follow-up requests.
Real World Insights (RWI) for Regulatory Submission
Building on the foundation of RWE for label expansion, Genomenon delivered Real World Insights (RWI)—evidence-backed interpretations connecting genotype, phenotype, therapy, and outcomes— tailored for Loxo’s regulatory dossier. This enabled Loxo to submit a defensible, actionable package to the PMDA and respond to follow-up inquiries with confidence.
Impact
Expand the Retevmo® label: Delivered a comprehensive, evidence-driven justification for including (and excluding) RET mutations on the label, ensuring the final list reflected only clinically meaningful variants.
Streamline regulatory submission: Enabled an on-time, actionable submission supported by a full body of real-world insights, literature references, and variant-level documentation.
Achieve regulatory success: Secured co-approval of Retevmo® and ODxTT for thyroid cancer in Japan, with an expanded label rooted in real-world patient benefit.
Accelerate time-to-approval: Compressed evidence generation timelines from months to weeks, allowing Loxo to meet a critical regulatory window without sacrificing rigor or completeness.
Strengthen future readiness: Created a scalable, literature-based evidence framework that can be re-applied for ongoing regulatory submissions and label expansions in other markets.
