NAMs Regulatory Case Studies
Real-world examples of successful NAMs regulatory submissions, qualifications, and acceptances across FDA, EMA, and global agencies.
Liver-on-Chip DILI Prediction
First organ-on-chip system data accepted in FDA IND submission for drug-induced liver injury prediction, reducing animal testing requirements.
Key Outcomes
- Data accepted in IND without additional animal studies
- 90%+ concordance with clinical hepatotoxicity
- Established precedent for OoC in drug development
Skin Sensitization IATA Qualification
EMA qualification opinion for defined approach combining multiple NAMs for skin sensitization assessment under OECD TG 497.
Key Outcomes
- Formal EMA qualification opinion published
- Valid across 27 EU member states
- Replaces Draize test for skin sensitization
PBPK Model for Pediatric Dosing
Physiologically-based pharmacokinetic modeling accepted as primary evidence for pediatric dose selection, avoiding additional clinical trials.
Key Outcomes
- Approved pediatric labeling based on PBPK
- No additional pediatric PK studies required
- Model-informed drug development precedent
Patient-Derived Tumor Organoids
Patient-derived tumor organoids used to support biomarker qualification and companion diagnostic development for oncology indication.
Key Outcomes
- Biomarker strategy supported by organoid data
- Precision medicine approach validated
- Companion diagnostic pathway established
HepaRG Hepatotoxicity Model
HepaRG cell line qualified by EMA for DILI prediction, providing standardized in vitro hepatotoxicity assessment across industry.
Key Outcomes
- EMA qualification opinion for DILI prediction
- Standardized cell model across sponsors
- Reduced reliance on primary hepatocytes
AI-Powered Toxicity Prediction
Machine learning model for cardiac toxicity prediction accepted in pre-IND meeting as supportive evidence for lead compound selection.
Key Outcomes
- AI model output accepted in regulatory discussion
- Reduced early-stage attrition risk
- Established AI/ML in drug development
"The FDA's acceptance of our organ-on-chip data represents a watershed moment for the industry. We've demonstrated that human-relevant NAMs can provide safety evidence that regulators trust, opening the door for broader adoption across drug development."