10 - drug discovery 2 Flashcards
1
Q
What LDL level would a patient with a PCKS9 gene mutation have?
A
<130 mg/dL
2
Q
What is the normal MOA of LDL-C regulation?
A
- PCSK9 is secreted
- PCKS9 binds to a LDL-R
- LDL particle binds to a PCSK9 bound LDL-R
- LDL is brought into the cell for lysosomal degradation
- LDL-R recycling is blocked
3
Q
What is the MOA of LDL-C regulation when a human monoclonal antibody against PCSK9 is introduced?
A
- The antibody binds to the PCSK9, which stops PCSK9 from binding to LDL-R
- LDL-R concentration is increased
- Without a bound LDL, the LDL-R is recycled
4
Q
Are PCSK9i considered a blockbuster drug at this moment?
A
No
5
Q
What is ICER?
A
Incremental cost effectiveness ratio
6
Q
What is QALY?
A
Quality adjusted life year
7
Q
How is ICER calculated?
A
ICER = (Cost2-cost1)/(QALY2-QALY1)
8
Q
How many estimated US adults might be eligible for PCSK9i if the cost is low enough?
A
Nearly 10 million
