Biologic product
a substance that is made from a living organism or its products and is used in the prevention, diagnosis, treatment, or cure of a disease.
First-in-class
drugs that possess a mechanism of action different from those of existing therapies (indicator of drug's degree of innovation)
Fast track
designated drugs have the potential to address unmet medical needs. Fast Track speeds new drug development and review.
Breakthrough Therapy
destination assigned to drugs with preliminary clonal evidence demonstrating that the drug may result in substantial improvement on at least one clinically significant endpoint over other available therapies for serious or life-threatening diseases for which there is unmet medical need.
Orphan diseases
affect fewer than 200,000 patient per year in the U.S
Phosphorylation
attachment of a phosphate group to an ion or molecule; important in activating enzymes
-ase
Protein therapeutics contain what stem suffix?
Monoclonal antibodies
large glycosylated proteins-produced from cell culture
-mab
Monoclonal antibodies contain what stem suffix?
MOA - monoclonal antibodies
(very specific) exert their effect on cell surface receptors, or scavenge proteins in extracellular matrix. Block signaling pathways.
AS-ON (Antisense Oligonucleotides)
synthetic nucleic acid based drugs that bind to specific mRNA strains.
-composed of short sequences; 13-25 nucleotides
-complementary to mRNA strands
-stop translation-> stops protein synthesis
-rsen
Which end suffix do Antisense Oligonucleotides contain?
Aptamers
RNA or DNA nucleic acid drugs that recognize specific proteins. The fold up into unique 3-D structures, allowing them to bind specifically to proteins.
-APT
Which suffix do most Aptamers contain?
siRNA
- therapeutics that target host mRNA
-commonly referred to as "kill the messenger"
-made from nucleic acids
Absorption, distribution, metabolism, elimination, toxicity
What does ADMET stand for?
Absorption
-movement of drug from site of application into bloodstream or target tissue
-measure of Bioavailability
Distribution
-movement of drug from blood to target site of action
Metabolism
Transformation of drug
Elimination
elimination of drug from body
Toxicity
Effect of drug on non-target tissues or cells
catabolized by proteases, water soluble, large MWT
Why are biologics NOT administered orally?
Bioavailability
amount of drug absorbed relative to quantity given
circulatory system
The fraction of oral drug that makes it to the ___ is the bioavailability of that drug
molecular weight, lipohilicity, number of HBD, number of HBA
What are Lipinski's Parameters for predicting Oral Bioavailability ?
<=500
What must the molecular weight be for a drug?
<=10
Number of Hydrogen Bond acceptors?
<=5
Number of hydrogen Bond donors?
0< Log P < 5
What must the log P be?
Enzymes, Monoclonal antibodies
Types of protein biologics
AS-ON, Aptamers, siRNA
Types of Nucleic Acid biologics
<= 5
What is the number of hydrogen bond donors to qualify for Lipinski's rule ?
<=10
What is the number of hydrogen bond acceptors to qualify for Lipniski's rule?