EXAM 1 - lecture 2: early stage drug discovery Flashcards
What best describes the field of medicinal chemistry?
The identification of a promising initial compound and its development into a drug with therapeutic properties
What is usually the first step in drug discovery?
Choose a disease
How do pharmaceutical companies choose what disease to target?
Since the pharmaceutical industry is a business, they want to choose a disease that will bring in money. They will likely target patient populations that are larger and have to be taken continuously = more sales = more $$.
What happens when a drug comes off patent?
When a drug comes off patent, other companies can make a generic form and after the generics hit the market, the brand name drugs will decline in sales bc they are too expensive
What makes it a generic drug?
The generic drug must work the same as the brand name and have the same active ingredient (API)
Differences allowed include color, shape, size, additives, binders, filling agents – excipients
Generics cost much less because the manufacturer didn’t spend the billions to discover the drug.
This is a reason why companies really want to find a way to extend patent lifetimes.
What age group are pharmaceutical companies trying to target now?
There has been an increase in the average life expectancy, which means more people are getting degenerative diseases/cancer that NEED drugs to improve well-being.
You can charge more for the drug because usually the patient doesn’t have any other options to choose from.
What group are pharmaceutical companies veering away from?
Cardiovascular diseases
There are so many cheap, effective medications that are already on the market that have already decreased the death rate. There are also different variations already made. Why would a patient want to switch from what already works?
What happened with Torcetrapib?
Statins (Lipitor) target HMG-CoA reductase, but when Pfizer was about to lose their patent, they planned to design a new way to regulate cholesterol - inhibit cholesterol ester transfer protein (CETP) which would decrease the amount of HDL to LDL.
Early clinical trials showed an increase in HDL and decrease in LDL but also a raise in blood pressure.
Eventually, the phase III trial (ILLUMINATE) showed significant increased risk of death bc of an unknown reason.
What disease is a pharmaceutical industry unlikely to develop a drug against?
A rare disease with small impacted population
What did companies do after hearing about Torcetrapib?
Other companies thought that the drug didn’t work because of the way it was compounded. They still believed inhibiting CETP would decrease LDL levels.
No company succeeded
What is an orphan drug/disease?
Treats a disease/disorder that affects a small portion of the population (<200,000 people in the US)
Usually rare genetic diseases
It is hard for companies to make the money back that they invested into the drug development
There are incentives given to make companies work on these hard-to-treat diseases
- government grants
- tax breaks
- extended market exclusivity
Alexion pharmaceuticals
Started developing drugs targeting the compliment system (auto-immune disorders)
Soliris was approved for treatment of paroxysmal nocturnal hemoglobinuria (destruction of RBCs by the complement system
Soliris was then approved for other complement system disorders
Costs $500k/yr for patients bc there are no other options
They didn’t even need to regain development costs bc 80% was publically funded
They were bought by AstraZeneca for $38B
