Biotech Glossary

An FDA pathway that allows drugs for serious conditions to be approved based on a surrogate endpoint (like tumor shrinkage) rather than clinical endpoints (like survival). The company must conduct post-approval studies to confirm benefit.

An FDA advisory committee meeting where external experts vote on whether a drug should be approved. While FDA is not required to follow the vote, they usually do. AdCom dates are major catalyst events for biotech stocks.

A certificate issued by a U.S. bank representing shares in a foreign company. Many European biotech companies trade in the US via ADRs (e.g., AstraZeneca as AZN, Novartis as NVS).

Application for approval of a generic drug. ANDAs do not require clinical trials proving safety and efficacy, only bioequivalence to the reference drug.

Demonstration that a generic drug has the same rate and extent of absorption as the brand-name drug. Required for ANDA approval.

Application submitted to FDA to request approval of a biologic product (proteins, antibodies, gene therapies). Similar to NDA but for biologics rather than small molecule drugs.

A biologic product highly similar to an already-approved reference biologic with no clinically meaningful differences. Approved via abbreviated pathway (351(k)).

FDA designation for drugs that show substantial improvement over existing treatments for serious conditions. Provides intensive FDA guidance, rolling review, and organizational commitment.

FDA order to stop a clinical trial, usually due to safety concerns. A partial clinical hold may allow some patients to continue while restricting new enrollment.

FDA letter indicating the application cannot be approved in its current form. The letter outlines deficiencies that must be addressed. Companies can resubmit or request a Type A meeting.

Company that provides clinical trial services to pharmaceutical and biotech companies, including patient recruitment, data management, and regulatory submission support.

Clinical trial design where neither patients nor investigators know who receives the treatment vs placebo. Reduces bias in outcome assessment.

Independent committee that monitors patient safety and treatment efficacy during a clinical trial. Can recommend stopping a trial early for safety concerns or overwhelming efficacy.

The regulatory agency responsible for drug approvals in the European Union. Equivalent to FDA in the US. Headquartered in Amsterdam.

The outcome measured in a clinical trial to evaluate whether a treatment works. Primary endpoints are the main measure of efficacy; secondary endpoints provide additional information.

FDA program for drugs treating serious conditions with unmet medical need. Provides more frequent FDA meetings, rolling review (submit sections as completed), and eligibility for Priority Review and Accelerated Approval.

U.S. federal agency responsible for approving drugs, biologics, and medical devices. The Center for Drug Evaluation and Research (CDER) handles most drug approvals.

SEC filing required when company insiders (executives, directors, 10% shareholders) buy or sell company stock. Filed within 2 business days of the transaction. Important signal for investors.

Regulations ensuring drugs are consistently produced and controlled according to quality standards. FDA inspects manufacturing facilities for GMP compliance.

Application submitted to FDA to begin clinical trials in humans. Contains preclinical data, manufacturing information, and clinical protocol. FDA has 30 days to review.

The specific disease or condition a drug is approved to treat. A drug may have multiple indications approved over time through label expansions.

Planned analysis of clinical trial data before the study is complete. May allow early stopping for efficacy, futility, or safety. Conducted by DSMB.

Approval of an already-marketed drug for a new indication, patient population, or dosage form. Often called a supplemental NDA or sBLA.

Probability estimate that a drug will receive FDA approval. Based on historical success rates by phase, therapeutic area, and other factors. Used for valuation models.

Application for drug approval submitted to EMA in Europe. Equivalent to NDA/BLA in the US.

Total value of a company's outstanding shares (share price × shares outstanding). Common size classifications: Micro (<$300M), Small ($300M-$2B), Mid ($2B-$10B), Large (>$10B).

How a drug works at the molecular level to produce its therapeutic effect. Important for understanding potential side effects and drug interactions.

Formal submission to FDA requesting approval to market a new drug. Contains all preclinical, clinical, and manufacturing data. FDA has 10-12 months to review (standard vs priority).

Special status for drugs treating rare diseases (<200,000 patients in US). Provides 7 years market exclusivity, tax credits, reduced fees, and FDA assistance. Popular biotech strategy.

Gold standard endpoint in oncology trials measuring time from treatment to death from any cause. Harder to achieve than PFS but more meaningful for patients.

Law requiring drug companies to pay fees to fund FDA drug review. In exchange, FDA commits to review timelines. PDUFA date is the deadline for FDA decision - a major catalyst event.

First-in-human studies testing safety, dosing, and pharmacokinetics in 20-100 healthy volunteers or patients. Success rate ~65%. Determines maximum tolerated dose.

Studies testing efficacy and side effects in 100-300 patients with the target disease. Success rate ~30%. Often divided into Phase 2a (dosing) and Phase 2b (efficacy).

Large pivotal studies comparing drug to standard of care in 300-3000+ patients. Success rate ~58%. Required for NDA submission. Often randomized, double-blind, placebo-controlled.

A company's portfolio of drugs in development across all stages (preclinical through Phase 3). Pipeline strength is a key valuation metric for biotech companies.

Inactive treatment (sugar pill, saline injection) given to control group in clinical trials. Allows measurement of true drug effect vs placebo effect.

Drug development stage before human testing. Includes laboratory studies (in vitro) and animal studies (in vivo) for safety and efficacy. Must complete before IND filing.

EMA scheme providing enhanced support for drugs addressing unmet medical needs. Similar to FDA Breakthrough Therapy. Offers early dialogue and accelerated assessment.

FDA designation reducing review time from 10 months to 6 months for drugs offering significant improvements over existing treatments. Often combined with other designations.

Time from treatment start until disease progression or death. Common endpoint in oncology trials. Easier to achieve than OS but may not reflect true patient benefit.

Gold standard study design where patients are randomly assigned to treatment or control groups. Minimizes bias and confounding variables. Required for most drug approvals.

FDA-required safety program for drugs with serious risks. May include medication guides, communication plans, or restricted distribution. Adds complexity and cost.

Ability to submit sections of an NDA/BLA as they are completed rather than all at once. Available with Fast Track and Breakthrough designations. Can shorten overall timeline.

Number of shares sold short (betting stock will decline) as percentage of float. High short interest can indicate bearish sentiment or set up a "short squeeze" on positive news.

Biomarker used as substitute for clinical outcome (e.g., tumor shrinkage instead of survival). Allows faster trials but may not reflect true patient benefit. Used in Accelerated Approval.

Disease category a drug targets. Major areas include: Oncology, CNS, Immunology, Cardiovascular, Infectious Disease, Rare Disease, and Metabolic.

Initial high-level clinical trial results, typically announcing whether primary endpoint was met. Full results with detailed data follow weeks/months later.

Condition with inadequate or no approved treatments. Drugs addressing unmet needs may qualify for expedited FDA programs and command premium pricing.