Acquired Orphan Blood Diseases Therapeutics Market Growth, Demand and Challenges of the Key Industry Players 2024-32
                     Global Acquired Orphan 
Blood Diseases 
Therapeutics Market 
Research and Forecast 
Report 2024-2032
Format: PDF+EXCEL
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In ternat ional  Market  Analys is  Research and Consul t ing Group is  a leading adv iser  on management  
s t rategy and market  research wor ldwide.  We par tner  wi th c l ients  in  a l l  regions and indust ry  
vert ica ls  to  ident i fy  thei r  h ighest -value oppor tuni t ies,  address thei r  most  cr i t ica l  chal lenges,  and 
t ransform thei r  bus inesses.
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are at  the top of  the company’s  exper t ise.
IMARC’s ta i lored approach combines unfathomable ins ight  in to the dynamics o f  companies and 
markets wi th c lose cooperat ion at  a l l  levels  of  the c l ient  organizat ion.  This ensures that  our  c l ients 
achieve unmatchable compet i t ive advantage,  bui ld  more pro f ic ient  organizat ions,  and secure 
las t ing resul ts .
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 .  I n  l i n e  w i t h  t h i s ,  t h e  s u d d e n  s h i f t  t o w a r d s  
v a l u e - b a s e d  h e a l t h c a r e  t h a t  i m p a c t s  r e i m b u r s e m e n t  p o l i c i e s ,  m a k i n g  i t  f i n a n c i a l l y  v i a b l e  f o r  h e a l t h c a r e  p r o v i d e r s  a n d  p a t i e n t s  t o  o p t  f o r  
a d v a n c e d  t r e a t m e n t  o p t i o n s ,  i s  c r e a t i n g  r e m u n e r a t i v e  g r o w t h  o p p o r t u n i t i e s  f o r  t h e  m a r k e t .
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
• Immunoglobul in Infusion Therapy
• Act ivated Prothrombin Complex Concentrate
• Thrombopoiet in Receptor Agonists
• Others
Report 
Disease Indication Insights:
Segmentation
• Acquired Agranulocytosis
• Acquired Hemophi l ia
• Acquired Von Wil lebrand Syndrome
• Paroxysmal Nocturnal  Hemoglobinuria (PNH)
• Myelodysplast ic Syndrome
• Others
Distribution Channel Insights:
• Hospital  Pharmacy
• Retai l  Pharmacy
• Others
Regional Insights:
Report 
• North America
Segmentation • Asia-Pacifi c
• Europe
• Latin America
• Middle East and Afr ica
• Alexion Pharmaceuticals Inc.  (AstraZeneca 
plc)
• Amgen Inc.
Competitive • GSK plc
Landscape • Novart is AG
• Novo Nordisk A/S
with Key • Otsuka Pharmaceutical  Co. Ltd.
Players • Rigel Pharmaceuticals Inc.
• 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?
What is the breakup of the market based on the 
distribution channel?
Key 
Questions Which is the most attractive distribution channel in the acquired orphan blood diseases therapeutics market?
Answered in What is the competitive structure of the global acquired 
orphan blood diseases therapeutics market?
the Report
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
Table of     7 . 2     A c q u i r e d  H e m o p h i l i a        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
Contents         7 . 3 . 1  M a r k e t  T r e n d s        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 :  
h t t p s : / / w w w. im a rcg ro u p . co m / a cq u i red - o r p h a n - b l o o d - d i s ea s es - t h
e r a p eu t i c s - m a r ke t / t o c
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