NeuroScience

Question 1

Vyvgart: generic name?

Answer 1

Efgartigimod alfa

Question 2

Efgartigimod: brand name?

Answer 2

Vyvgart

Question 3

Vyvgart: indication?

Answer 3

Seropositive (acetylcholine-receptor antibody-positive) generalized myasthenia gravis (gMG)

Question 4

Vyvgart: conditions for NHB?

Answer 4

After treatment failure with both IVIG and rituximab

Question 5

Vyvgart: conditions for private insurance?

Answer 5

After treatment failure with either IVIG or rituximab

Question 6

Vyvgart: mechanism of action?

Answer 6

Neonatal Fc receptor (FcRN) blocker, thus reducing total level of IgG

Question 7

Vyvgart: how is it administered?

Answer 7

IV, once weekly for 4 weeks. May repeat cycles.

Question 8

Vyvgart: partner?

Answer 8

Argenx

Question 9

Spinraza: generic name?

Answer 9

Nusinersen

Question 10

Nusinersen: brand name?

Answer 10

Spinraza

Question 11

Spinraza: partner?

Answer 11

Biogen

Question 12

Spinraza: indication(s)?

Answer 12

Spinal Muscular Atrophy (SMA)

Question 13

Spinraza: mechanism of action?

Answer 13

Antisense Oligonucleotide (ASO) that increases production of SMN2, thus compensating for the mutated SMN1 gene

Question 14

Kyprolis: generic name?

Answer 14

Carfilzomib

Question 15

Carfilzomib: brand name?

Answer 15

Kyprolis

Question 16

Kyprolis: indication(s)?

Answer 16

Relapsed or refractory multiple myeloma, in patients who’ve received previous treatments.
Kyprolis is given in combination with other treatments.

Question 17

Kyprolis: partner?

Answer 17

Amgen

Question 18

Kyprolis: mechanism of action?

Answer 18

Proteasome inhibitor. As a result, protein is accumulated in the myeloma cell and the cell dies.

Question 19

Uplizna: generic name?

Answer 19

Inebilizumab

Question 20

Inebilizumab: brand name?

Answer 20

Uplizna

Question 21

Uplizna: indication(s)?

Answer 21

Aquaporin-4-IgG+ Neuromyelitis Optica Spectrum Disorder (NMOSD)

Question 22

Uplizna: partner?

Answer 22

Amgen

Question 23

Zurzuvae: generic name?

Answer 23

Zuranolone

Question 24

Zuranolone: brand name?

Answer 24

Zurzuvae

Question 25

Zuranolone: indication(s)

Answer 25

Post-partum depression

Question 26

Zuranolone: partner?

Answer 26

Biogen

Question 27

Zuranolone: administration?

Answer 27

PO, 50 mg once daily for 14 days

Question 28

Zuranolone: mechanism of action?

Answer 28

GABA-A receptor positive allosteric modulator (PAM), enhancing signalling through the receptor

Question 29

Voxzogo: generic name?

Answer 29

Vosoritide

Question 30

Vosoritide: Brand name?

Answer 30

Voxzogo

Question 31

Voxzogo: partner?

Answer 31

BioMarin

Question 32

Voxzogo: Indication(s)?

Answer 32

Achondroplasia and open growth plates

Question 33

Voxzogo: Administration?

Answer 33

Daily S/C injection

Question 34

Voxzogo: Adverse effect(s)?

Answer 34

Decrease in blood pressure

Question 35

Voxzogo: mechanism of action?

Answer 35

In achondroplasia, bone growth is inhibited due to a mutation in Fibroblast Growth Factor Receptor 3 (FGFR3). Vosorotide antagonizes downstream signalling in the receptor's signal pathway and promotes chondrocytes proliferation and differentiation.

Question 36

Uplizna (Inebilizumab): Administration?

Answer 36

Initial: Two IV infusions over 2 weeks Maintenance: every 6 months

Question 37

Daybue: generic name?

Answer 37

Trofinetide

Question 38

Trofinetide: brand name?

Answer 38

Daybue

Question 39

Daybue: indication(s)?

Answer 39

Rett syndrome

Question 40

Daybue: administration

Answer 40

PO

Question 41

Daybue: partner?

Answer 41

Acadia