A.32

Question 1

advanced therapy medicinal products (ATMPs)

Answer 1

Medicines for human use that are based on genes, tissues or cells; offer ground-breaking new opportunities for the treatment of disease and injury

Question 2

biological medicine products

Answer 2

medicine containing biological material, macromolecules produced by or extracted from a biological source

Question 3

biological drugs

Answer 3

-biological drugs cannot be fully characterized due to the structural complexity of the macromolecules
- We need to use a combination of physiochemical biological testing, together with process control, to define their quality and characteristics

Question 4

chemical medicinal products

Answer 4

• medicines containing chemical active ingredients
• small molecules which can be accurately characterized and reproduced chemicall

Question 5

examples of advanced therapy medicinal products (ATMPs)

Answer 5

• gene therapy medicines
• somatic cell therapy medicines
• tissue engineered medicines

Question 6

gene therapy medicines

Answer 6

• contain genes that lead to a therapeutic, prophylactic or diagnostic effect
• work by inserting ‘recombinant’ genes into the body, usually to treat a variety of diseases including genetic disorders, cancer or long term diseases
• eg. for multiple myeloma or spinal muscular atrophy

Question 7

orphan drugs

Answer 7

medicinal products intended for the diagnosis, prevention or treatment of super rare diseases (→ affect less than 1/2000 people)

Question 8

somatic cell therapy medicines

Answer 8

• contains cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential function of the body
• eg. used for cystic fibrosis

Question 9

tissue engineering medicines

Answer 9

• contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissues
• eg. artificial skin and cartilage

Question 10

types of biological drugs

Answer 10

• vaccines
• blood components
• cells
• allergens
• genes
• tissues
• antitoxins
• antibodies
• recombinant proteins

Question 11

why are orphan drugs called orphan drugs?

Answer 11

These drugs are called “orphan” because under normal market conditions the pharmaceutical industry has little interest in developing and marketing products intended for only a small number of patients.