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Medicinal Chemistry > Hemophillia > Flashcards

Hemophillia Flashcards

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1
Q

What is hemophilia?

A

Genetically inherited bleeding disorder

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2
Q

What genes are affected and where is it located?

A

Genes for factor VIll and IX located on the x chromosome

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3
Q

What is the root cause for hemophilia

A

Low or no production of particular clotting factors

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4
Q

What are the types of hemophilia

A

Hemophilia A: factor VIll deficiency

Hemophilia B: factor IX deficiency

Acquired hemophilia: autoantibody against VIII

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5
Q

What are hemophilia complications?

A

Joint damage/disease

Risk for viral infections

Inhibitor/ neutralizing antibodies

Heart disease, high blood pressure

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6
Q

Joint damage/ disease

A

Chronic bleeding at joints

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7
Q

Risk of viral infections

A

HIV

Hepatitis

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8
Q

Inhibitor/ neutralizing antibodies

A

30% gf hemophilia A patients develop abs

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9
Q

Heart disease/HBP

A

Caused partly by inability to be active

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10
Q

Hemophilia therapeutics

A

Plasma transfusion of low concentrations of clotting factors

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11
Q

How was plasma concentration of clotting factors improved

A

Cryopercipitation

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12
Q

What was the major concern for increased plasma concentration of clotting factors through cryopercipitation

A

Neutralizing antibodies produced in the body

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13
Q

Cryoprecipitate factor VIll

A

Treat hemophilia A

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14
Q

What is the major concern for the use of cryoprecipitate factor VIll

A

Caries risk of viral infection

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15
Q

How is cryoprecipitate factor VIll prepared

A

From a single donor and whole blood

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16
Q

Why was cryoprecipitate factor VIll preferred over lypholized

A

Lypholization disrupts the polymeric structure of factor VIll and it requires pooling from multiple homers

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17
Q

Lyophilized factor IX

A

Treatment of hemophilia B

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18
Q

How is lyophilized factor IX prepared

A

Extracted from plasma, most inactive protein but can still activate clotting factors

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19
Q

Why is heparin added for patients taking lyophilized factor IX

A

To limit thrombin overactivation

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20
Q

What is mononine?

A

Monoclonal antibody specific to factor IX to cleanly isolate the targeted factor IX which is separated from antibody and undergoes further purification

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21
Q

How is monomine prepared and administered?

A

Pooled from human plasma and administered via IV

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22
Q

Which patient is administered NovoSeven (rFactor VIIa)

A

Patient with VIl deficiency and treatment of bleeding episodes in hemophilia

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23
Q

What is the MOA of NovoSeven

A

Increases thrombin activity activity for both type A and B

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24
Q

How is NovoSeven prepared?

A

Human gene recombinantly expressed in baby hamster kidney cells

Secreted protein is cleaved to its VIIa active form

Packaged in lyophilized single use vial (IV)

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25
Q

What is NoveSeven RT

A

Room temperature stable formulation of NovoSeven

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26
Q

Obizur (r porcine factor VIll): what type of hemophilia is it used for?

A

A

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27
Q

Obizur is what kind of therapeutic

A

Antibody with a 90 kD heavy chain and 80 kD light chain

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28
Q

How is obizur produced?

A

Expressed in hamster kidney cells and affinity for purified

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29
Q

Obizur half life

A

11 hours

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30
Q

How is obizur packaged and administered

A

Packaged lyophilized

Administered IV

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31
Q

True or false: obizur has a comparable activity to human factor VIII

A

True

32
Q

Eloctate (r Factor VIII- Fc fusion protein) is approved for the treatment of what type of hemophilia

A

A

33
Q

What type of therapeutic is eloctate?

A

Analog of hFactor VIII covalently linked to human lgG

34
Q

When the IgG region of eloctate binds to an IgG receptor, what occurs?

A

There is a delay in lysosomal degradation of IgG by returning them to circulation

35
Q

Eloctate half life

A

16-20hrs

36
Q

How is eloctate produced?

A

Expressed in HEK cells and purified

37
Q

How is eloctate-packaged and administered

A

lyophilized and IV

38
Q

Nuwiq (r human factor VIIII; truncated) is approved for the treatment of what type of hemophilia

A

A

39
Q

What is the characteristic of nuwiq

A

Contains heavy and light chain that lacks a b-domain

40
Q

True or false: Nuwiq heavy and light chain not are covalently attached

A

True

41
Q

Nuwiq half life

A

17 hours

42
Q

How is Nuwiq produced

A

HEK cells and purified

43
Q

How’s Nuwiq packaged and administered

A

lyophilized and IV

44
Q

Kovaltry (r human factor VIll) is approved for what type of hemophilia?

A

A

45
Q

What type of therapeutic is kovaltry?

A

Unmodified full length human factor VIII

46
Q

How is kovaltry produced?

A

Express in hamster kidney cells and co-expressed with HSP70 proper folding

47
Q

Kovaltry half life

A

12 hrs

48
Q

How is kovaltry packaged and administered?

A

Lyophilized and IV

49
Q

Aystyla (r single chain factor VIII) is approved for what type of hemophilia

A

A

50
Q

How’s afstyla designed

A

Single chain with heavy and light chain covalently linked

51
Q

What clotting factor does afstyla increases its stability and has affinity for?

A

Von willebrand factor

52
Q

What is the function of Von willebrand factor?

A

Prevents degradation of factor VIII

53
Q

What other uses are there for afstyla?

A

Hemophilia A bleeding episodes

Routine prophylaxis

preop treatment

54
Q

Afstyla half life

A

14 hours

55
Q

How is afstyle prepared and administered

A

Lyophilized and IV

56
Q

How is hemlibra (emicizumab) used in hemophilia theragry

A

It is a bispecific antibody that targets factor IXa and X and used as prophylaxis to reduce hemophilia A bleeding events, for patients with or without factor VIll inhibitors

57
Q

What are other uses of hemlibra

A

Swelling and joint pain

58
Q

What is the black box warning for hemlibra

A

Thrombotic microangiopathy and thrombotic events

59
Q

What is esperoct (anti-hemophilic factor, glyopegylated- exei) used for

A

Hemophilia A

60
Q

What is esperoct

A

Factor 8 analog linked to PEG (40 kD), used to prevent or reduce bleeding episodes of hemophilia A

61
Q

Why does esperoct have PEG

A

It increases half life and reduces clearance

62
Q

Esperoct half life

A

21.7 hrs

63
Q

Aprolix (Factor IX-Fc fusion protein) use?

A

Hemophilia B

64
Q

What type of therapeutic is alprolix

A

Recombinant factor IX fused to Fc fusion protein (IgG)

Delays degradation and increases half-life

65
Q

How is alprolix produced?

A

Expressed in HEK cells and purified

66
Q

Alprolix half life

A

86 hours

67
Q

Idelvion (r Factor IX fused to albumin) hemophilia type

A

B

68
Q

Idelvion therapeutic type

A

Genetic fusion of human factor IX and human

albumin; expressed as a single protein

69
Q

Idelvion mechanisms action

A

Circulates as fusion protein until factor IX is
activated
Albumin is cleaved from factor IX only
when it is needed for clotting

70
Q

Idelvion half life

A

90 hours

71
Q

Rebinyn (Factor IX, GlycoPEGylated) Hemophilia type

A

B

72
Q

Rebinyn (Factor IX, GlycoPEGylated) therapeutic type

A

Factor IX is glycoPEGylated to increase

circulating half-life of Factor IX

73
Q

Rebinyn (Factor IX, GlycoPEGylated) Half life

A

118 hours

74
Q

Rebinyn (Factor IX, GlycoPEGylated) Modification causes

A

Lower bleeding frequency and less frequent IV dosing

75
Q

What is the only reconstituted IV

A

Idelvion (r Factor IX fused to albumin)

Medicinal Chemistry (2 decks)

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