Acquired Orphan Blood Diseases Therapeutics Market by Product Type, Distribution Channel, End User 2024-2032
                     Global Acquired Orphan 
Blood Diseases Therapeutics 
Market Research and 
Forecast Report 2024-2032
Format: PDF+EXCEL
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i ts  c l ients ’  bus iness pr ior i t ies  and del iver ing ta i lored solut ions that  dr ive meaningfu l  outcomes.  Our 
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A c c o r d i n g  t o  t h e  l a t e s t  r e p o r t  b y  I M A R C  G r o u p ,  t i t l e d  " A c q u i r e d  O r p h a n  B l o o d  D i s e a s e s  
T h e r a p e u t i c s  M a r k e t :  G l o b a l  I n d u s t r y  T r e n d s ,  S h a r e ,  S i z e ,  G r o w t h ,  O p p o r t u n i t y  a n d  
F o r e c a s t  2 0 2 4 - 2 0 3 2 , "  t h e  g l o b a l  a c q u i r e d  o r p h a n  b l o o d  d i s e a s e s  t h e r a p e u t i c s  m a r k e t  s i z e  
r e a c h e d  U S $  8 . 8  B i l l i o n  i n  2 0 2 3 .
A c q u i r e d  o r p h a n  b l o o d  d i s e a s e  t h e r a p e u t i c s  r e f e r s  t o  t h e  s p e c i a l i z e d  m e d i c a l  t r e a t m e n t s  
Report d e s i g n e d  f o r  r a r e  b l o o d  d i s o r d e r s  t h a t  a r e  d e v e l o p e d  p o s t - b i r t h .  T h e y  i n c l u d e  a  r a n g e  o f  t r e a t m e n t  m o d a l i t i e s ,  s u c h  a s  t a r g e t e d  t h e r a p i e s ,  b i o l o g i c s ,  s m a l l  m o l e c u l e  i n h i b i t o r s ,  a n d  
g e n e  t h e r a p i e s .  A c q u i r e d  o r p h a n  b l o o d  d i s e a s e  t h e r a p e u t i c s  a r e  e n g i n e e r e d  t o  c o m b a t  
Highlight and s p e c i f i c  a s p e c t s  o f  t h e  d i s o r d e r  b y  u t i l i z i n g  c o m p l e x  b i o l o g i c a l  c o m p o n e n t s ,  p r o t e i n s ,  o r  s y n t h e t i c  m o l e c u l e s .  T h e y  a r e  e m p l o y e d  i n  h o s p i t a l s ,  s p e c i a l i z e d  t r e a t m e n t  c e n t e r s ,  a n d  
r e s e a r c h  i n s t i t u t i o n s .  
Description A c q u i r e d  o r p h a n  b l o o d  d i s e a s e  t h e r a p e u t i c s  o f f e r  n u m e r o u s  b e n e f i t s ,  i n c l u d i n g  e a r l y  i n t e r v e n t i o n ,  p e r s o n a l i z e d  t r e a t m e n t  p l a n s ,  i m p r o v e d  p a t i e n t  q u a l i t y  o f  l i f e ,  m i n i m i z e d  d i s e a s e  
p r o g r e s s i o n ,  a n d  c u r a t i v e  p o s s i b i l i t i e s .  A d d i t i o n a l l y ,  t h e y  o f f e r  r e d u c e d  s i d e  e f f e c t s ,  t a r g e t e d  
d r u g  d e l i v e r y ,  q u i c k e r  r e c o v e r y ,  c o s t - e f f e c t i v e n e s s  i n  l o n g - t e r m  d i s e a s e  m a n a g e m e n t ,  a n d  a n  
i n c r e a s e d  t r e a t m e n t  s u c c e s s  r a t e .
R e q u e s t  f o r  a  P D F  s a m p l e  o f  t h i s  r e p o r t :  
h t t p s : / / w w w . i m a r c g r o u p . c o m / a c q u i r e d - o r p h a n - b l o o d - d i s e a s e s - t h e r a p e u t i c s - m a r k e t / r e q u e s t s a m
p l e
Report Description
G lo b a l  A c q u i r e d  O r p h a n  B l o o d  D is e a s e s  T h e r a p e u t i c s  M a r k e t  T r e n d s :
T h e  r a p i d  a d v a n c e m e n t s  i n  m e d i c a l  r e s e a r c h  c o n t r i b u t i n g  t o  t h e  i d e n t i f i c a t i o n  a n d  d e v e l o p m e n t  o f  e f f e c t i v e  a n d  t a r g e t e d  t r e a t m e n t s  f o r  
a c q u i r e d  o r p h a n  b l o o d  d i s e a s e s ,  a r e  m a j o r  f a c t o r s  p r o p e l l i n g  t h e  m a r k e t  g r o w t h .  M o r e o v e r ,  t h e  i m p l e m e n t a t i o n  o f  g o v e r n m e n t  p o l i c i e s  a n d  
i n c e n t i v e s ,  s u c h  a s  e x c l u s i v e  m a r k e t i n g  r i g h t s  a n d  t a x  c r e d i t s  t h a t  e n c o u r a g e  p h a r m a c e u t i c a l  c o m p a n i e s  t o  i n v e s t  i n  r e s e a r c h  a n d  
i n n o v a t i o n  i n i t i a t i v e s ,  i s  s u p p o r t i n g  t h e  m a r k e t  g r o w t h .  A l o n g  w i t h  t h i s ,  t h e  g r o w i n g  a w a r e n e s s  a n d  a d v o c a c y  e f f o r t s  b y  p a t i e n t  
o r g a n i z a t i o n s  a n d  h e a l t h c a r e  p r o v i d e r s  t o  e d u c a t e  t h e  p u b l i c  a n d  p o l i c y - m a k e r s  a b o u t  t h e  c r i t i c a l  n e e d  f o r  t r e a t m e n t  i s  s t i m u l a t i n g  t h e  
m a r k e t  g r o w t h .  B e s i d e s  t h i s ,  r a p i d  a d v a n c e s  i n  g e n o m i c  a n d  p r o t e o m i c  t e c h n o l o g i e s  l e a d i n g  t o  t h e  p r e c i s e  i d e n t i f i c a t i o n  o f  d i s e a s e  
e t i o l o g i e s  a r e  a n t i c i p a t e d  t o  d r i v e  t h e  m a r k e t  g r o w t h .  F u r t h e r m o r e ,  t h e  r i s i n g  p a r t n e r s h i p s  a n d  c o l l a b o r a t i o n s  a m o n g  r e s e a r c h  i n s t i t u t i o n s ,  
p h a r m a c e u t i c a l  c o m p a n i e s ,  a n d  h e a l t h c a r e  p r o v i d e r s  f o r  s h a r i n g  k n o w l e d g e ,  r e s o u r c e s ,  a n d  f i n a n c i a l  r i s k s  t h a t  a c c e l e r a t e  t h e  
d e v e l o p m e n t  a n d  d i s t r i b u t i o n  o f  t h e r a p e u t i c s  a r e  b o l s t e r i n g  t h e  m a r k e t  g r o w t h .  I n  a d d i t i o n  t o  t h i s ,  t h e  i n c r e a s i n g  e s t a b l i s h m e n t  o f  
s p e c i a l i z e d  h e a l t h c a r e  f a c i l i t i e s  a n d  t h e  a v a i l a b i l i t y  o f  s k i l l e d  p r o f e s s i o n a l s  t h a t  s e r v e  t o  d e c e n t r a l i z e  t r e a t m e n t ,  m a k i n g  i t  m o r e  
a c c e s s i b l e  t o  a  b r o a d e r  p a t i e n t  b a s e ,  i s  a c t i n g  a s  a  g r o w t h - i n d u c i n g  f a c t o r .  A p a r t  f r o m  t h i s ,  t h e  r i s i n g  p r e v a l e n c e  o f  p e r s o n a l i z e d  m e d i c i n e  
a n d  t h e  g r o w i n g  f o c u s  o n  p a t i e n t - c e n t r i c  c a r e  m o d e l s  a r e  a l s o  f a v o r i n g  t h e  m a r k e t  g r o w t h .
L o o k in g  f o r w a r d ,  t h e  m a r k e t  v a lu e  i s  p r o j e c t e d  t o  r e a c h  U S $  1 7 .2  B i l l i o n  b y  2 0 3 2 ,  e x p a n d in g  a t  a  C A G R  o f  7 .5 %  d u r in g  
2 0 2 4 - 2 0 3 2 .
V ie w  R e p o r t  T O C ,  F i g u r e s  a n d  T a b l e s :  h t t p s : / / w w w . i m a r c g r o u p . c o m / a c q u i r e d - o r p h a n - b lo o d - d is e a s e s - t h e r a p e u t i c s - m a r k e t
Therapy Insights:
• Recombinant Factor
• Immunoglobulin Infusion Therapy
• Activated Prothrombin Complex Concentrate
• Thrombopoietin Receptor Agonists
• Others
Report 
Segmentation Disease Indication Insights:
• Acquired Agranulocytosis
• Acquired Hemophil ia
• Acquired Von Wil lebrand Syndrome
• Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Myelodysplast ic Syndrome
• Others
Distribution Channel Insights:
• Hospital  Pharmacy
• Retai l  Pharmacy
• Others
Report Regional Insights:
• North America
Segmentation
• Asia-Pacifi c
• Europe
• Latin America
• Middle East and Afr ica
• Alexion Pharmaceuticals Inc.  (AstraZeneca 
plc)
• Amgen Inc.
• GSK plc
Competitive • Novart is AG
Landscape • Novo Nordisk A/S
with Key • Otsuka Pharmaceutical  Co. Ltd.
• Rigel Pharmaceuticals Inc.
Players • Roche Holding AG
• Sanofi
• Takeda Pharmaceutical  Company Limited
How has the global acquired orphan blood diseases therapeutics 
market performed so far, and how will it perform in the coming 
years?
What are the drivers, restraints, and opportunities in the global 
acquired orphan blood diseases therapeutics market?
Key What is the impact of each driver, restraint, and opportunity on the 
global acquired orphan blood diseases therapeutics market?
Questions 
Answered in What are the key regional markets?
the Report
Which countries represent the most attractive acquired orphan 
blood diseases therapeutics market?
What is the breakup of the market based on the therapy?
Which is the most attractive therapy in the acquired orphan blood 
diseases therapeutics market?
What is the breakup of the market based on the disease indication?
Which is the most attractive disease indication in the acquired 
orphan blood diseases therapeutics market?
Key What is the breakup of the market based on the distribution 
channel?
Questions 
Which is the most attractive distribution channel in the acquired 
Answered in orphan blood diseases therapeutics market?
the Report What is the competitive structure of the global acquired orphan 
blood diseases therapeutics market?
Who are the key players/companies in the global acquired orphan 
blood diseases therapeutics market?
1     P r e f a c e
2     S c o p e  a n d  M e t h o d o l o g y
    2 . 1     O b j e c t i v e s  o f  t h e  S t u d y
    2 . 2     S t a k e h o l d e r s
    2 . 3     D a t a  S o u r c e s
        2 . 3 . 1     P r i m a r y  S o u r c e s
        2 . 3 . 2     S e c o n d a r y  S o u r c e s
    2 . 4     M a r k e t  E s t i m a t i o n
Table of         2 . 4 . 1     B o t t o m - U p  A p p r o a c h        2 . 4 . 2     T o p - D o w n  A p p r o a c h
    2 . 5     F o r e c a s t i n g  M e t h o d o l o g y
3     E x e c u t i v e  S u m m a r y
4     I n t r o d u c t i o n
Contents     4 . 1     O v e r v i e w    4 . 2     K e y  I n d u s t r y  T r e n d s5     G l o b a l  A c q u i r e d  O r p h a n  B l o o d  D i s e a s e s  T h e r a p e u t i c s  M a r k e t
    5 . 1     M a r k e t  O v e r v i e w
    5 . 2     M a r k e t  P e r f o r m a n c e
    5 . 3     I m p a c t  o f  C O V I D - 1 9
    5 . 4     M a r k e t  F o r e c a s t
6      M a r k e t  B r e a k u p  b y  T h e r a p y
    6 . 1     R e c o m b i n a n t  F a c t o r
        6 . 1 . 1  M a r k e t  T r e n d s
        6 . 1 . 2  M a r k e t  F o r e c a s t
    6 . 2     I m m u n o g l o b u l i n  I n f u s i o n  T h e r a p y
        6 . 2 . 1  M a r k e t  T r e n d s
        6 . 2 . 2  M a r k e t  F o r e c a s t
    6 . 3     A c t i v a t e d  P r o t h r o m b i n  C o m p l e x  C o n c e n t r a t e
        6 . 3 . 1  M a r k e t  T r e n d s
        6 . 3 . 2  M a r k e t  F o r e c a s t
       
    6 . 4     T h r o m b o p o i e t i n  R e c e p t o r  A g o n i s t s
        6 . 4 . 1  M a r k e t  T r e n d s
        6 . 4 . 2  M a r k e t  F o r e c a s t
    6 . 5     O t h e r s
        6 . 5 . 1  M a r k e t  T r e n d s
        6 . 5 . 2  M a r k e t  F o r e c a s t
7    M a r k e t  B r e a k u p  b y  D i s e a s e  I n d i c a t i o n
    7 . 1     A c q u i r e d  A g r a n u l o c y t o s i s
        7 . 1 . 1  M a r k e t  T r e n d s
        7 . 1 . 2  M a r k e t  F o r e c a s t
    7 . 2     A c q u i r e d  H e m o p h i l i a
Table of         7 . 2 . 1  M a r k e t  T r e n d s        7 . 2 . 2  M a r k e t  F o r e c a s t
    7 . 3     A c q u i r e d  V o n  W i l l e b r a n d  S y n d r o m e
        7 . 3 . 1  M a r k e t  T r e n d s
Contents         7 . 3 . 2  M a r k e t  F o r e c a s t
    7 . 4     P a r o x y s m a l  N o c t u r n a l  H e m o g l o b i n u r i a  ( P N H )
        7 . 4 . 1  M a r k e t  T r e n d s
        7 . 4 . 2  M a r k e t  F o r e c a s t
    7 . 5     M y e l o d y s p l a s t i c  S y n d r o m e
        7 . 5 . 1  M a r k e t  T r e n d s
        7 . 5 . 2  M a r k e t  F o r e c a s t
    7 . 6     O t h e r s
        7 . 6 . 1  M a r k e t  T r e n d s
        7 . 6 . 2  M a r k e t  F o r e c a s t
8    M a r k e t  B r e a k u p  b y  D i s t r i b u t i o n  C h a n n e l
    8 . 1     H o s p i t a l  P h a r m a c y
        8 . 1 . 1  M a r k e t  T r e n d s
        8 . 1 . 2  M a r k e t  F o r e c a s t     
F o r  m o r e  i n f o r m a t i o n ,  v i s i t :  
ht t ps : / /w ww. i ma rc gro up . c o m /a cq u i red - o rph a n -b l o o d -d i sea se s
- t he ra peut i c s -ma rke t / t o c
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