macromolecular drugs questions from roldo

Question 1

What advantages can be gained by maximising systemic bioavailability of a drug after oral or transmucosal administration?

Answer 1

needle-free delivery so increased patient acceptance and improved compliance

reduction of intra- and inter-patient variability, thus improving the control of the drug’s intended and unintended actions. (Low bioavailability has been shown to be associated with large inter-subject variability in systemic exposure.)

economic advantage of reducing the waste of the drug.

Question 2

What types of molecules would need an absorption enhancer if administered orally or transmucosally?
Explain why these molecules present poor absorption?

Answer 2

Proteins and peptides used chronically or that require frequent dosing (i.e. insulin, calcitonin)
Non-peptide macromolecules (e.g. heparin)
Oligonucleotides

Question 3

. What are the factors that affect mucoadhesion?

Answer 3

Molecular weight
Flexibility
Cross-linking density
Hydrogen-bonding capacity
Hydration
Charge
Concentration

Question 4

What types of mucoadhesive dosage forms can be developed?

Answer 4

Tablets
Films
Patches
Gels/ointments

Question 5

What are the delivery routes that can benefit from the use of mucoadhesive delivery systems?

Answer 5

Buccal
Sublingual
Vaginal nasal
eye

Question 6

what is the indication for Aldurazyme 100 U/ml concentrate for solution for infusion?

Answer 6

Aldurazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Mucopolysaccharidosis I (MPS I; α-L-iduronidase deficiency) to treat the non-neurological manifestations of the disease

Question 7

what is the content in Aldurazyme 100 U/ml concentrate for solution for infusion?

Answer 7

1 ml contains 100 U (approximately 0.58 mg) of laronidase.

Question 8

what are the excipients of Aldurazyme 100 U/ml concentrate for solution for infusion

Answer 8

Sodium chloride

Sodium phosphate monobasic, monohydrate

Sodium phosphate dibasic, heptahydrate

Polysorbate 80

Water for injections

Question 9

what are the ingredients of pfizer mRNA vaccine?

Answer 9

mRNA

potassium chloride

monobasic potassium

dibasic sodium phosphate dhydrate

sodium chloride

sucrose

Question 10

what mRNA is used in in the vaccine?

Answer 10

4-hydroxybutyl)azanediyl)bis(hexane-6,1-diyl)bis(2-hexyldecanoate)

2 [(polyethylene glycol)-2000]-N,N-ditetradecylacetamide

1,2-Distearoyl-sn-glycero-3- phosphocholine
cholesterol