Therapeutics Exam 1

Small Molecules (Therapeutic Classification)

Drugs with a molecular weight MW ≤ 1000 g/mol. They have relatively simple structures and are typically orally bioavailable (taken as pills/tablets).

Macromolecules (Therapeutic Classification)

Drugs with a molecular weight MW > 1000 g/mol (e.g., proteins, antibodies, nucleic acids, peptides). Delivered parenterally (via injection) because they cannot survive or cross the GI tract.

Enteral vs. Parenteral Administration

Enteral: Via the GI tract (Oral, Sublingual, Buccal, Rectal). Oral is the preferred goal route. Parenteral: Bypasses the GI tract (IV, Subcutaneous, Epidural, Insufflation).

Active Pharmaceutical Ingredient (API)

The specific active molecule in a drug formulation that produces the therapeutic effect (e.g., small molecule, antibody, RNA).

Excipients

All inactive components in a drug formulation besides the API. They aid in stability, solubility, flavor, color, metabolism, delivery, storage, and controlled release.

Agonist vs. Antagonist

Agonist: Binds to a receptor and stimulates/activates a response. Antagonist: Binds to a receptor and inhibits/prevents a response.

CDER (Center for Drug Evaluation and Research)

The branch of the FDA responsible for reviewing drug applications and setting standards for drug safety and efficacy.

IND (Investigational New Drug) Application

An application submitted to the FDA requesting permission to test a drug in humans following promising preliminary/preclinical testing.

NDA (New Drug Application)

An application submitted to the FDA to gain approval to bring a drug to market following successful human clinical trials.

First-in-Class

A drug that works through a novel Mechanism of Action (MoA). In practice, pharmaceutical companies often use this as a marketing term for drugs with only minor variations.

First-in-Indication

The very first approved drug brought to market to treat a specific disease or condition (less common, usually seen in rare/orphan or newly discovered diseases).