Primary Endpoint
The main result measured in a trial to determine whether the treatment works, defined before the trial begins.
Biotech & FDA Glossary
Key terms for understanding clinical-stage biotech investing
Phase 1
First-in-human trials testing safety and dosage in a small group, typically 20-80 participants.
Phase 2
Trials assessing efficacy and side effects in a larger group, usually 100-300 participants.
Phase 3
Large-scale trials (300-3,000+) confirming efficacy and monitoring adverse reactions before approval.
PDUFA Date
The Prescription Drug User Fee Act deadline by which the FDA must review and decide on a new drug application.
NDA / BLA
New Drug Application / Biologics License Application - the formal request for FDA approval to market a drug or biologic.
Primary Completion Date
The date when the last participant is examined for the primary endpoint - often the key catalyst for investors.
Indication
The specific disease or condition a drug is being developed to treat.
Breakthrough Therapy
An FDA designation that expedites development of drugs showing substantial improvement over existing options.
Fast Track
An FDA process designed to speed review of drugs treating serious conditions with unmet medical need.