Pompe Disease Treatment Market by Product Type, Distribution Channel, End User 2024-2032
                     Global Pompe Disease 
Treatment Market 
Research and Forecast 
Report 2024-2032
Format: PDF+EXCEL
© 2023 IMARC All Rights Reserved 
About IMARC Group
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.
IMARC’s in format ion products  inc lude major market ,  sc ient i f ic ,  economic and technological  
developments  for  bus iness leaders  in  pharmaceut ica l ,  indust r ia l ,  and h igh technology 
organizat ions.  Market  forecasts  and indust ry  analys is  for  b iotechnology,  advanced mater ia ls ,  
chemicals ,  food and beverage,  t ravel  and tour ism, nanotechnology and novel  process ing methods 
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 .
Accord ing  t o  the  la tes t  r epo r t  by  IM ARC Group,  t i t l ed  " Pompe Disease 
T reatment  Market :  G l oba l  Indust ry  T rends,  Share ,  S ize ,  Growth ,  Oppor tun i ty  
and  Forecast  2024-2032 ,"  t he  g loba l  Pompe  d isease t rea tment  marke t  s i ze  
reached  US$  1 .0  B i l l i on  i n  2023.
Report Factors Af fect ing  the  Growth  of  the Pompe Disease Treatment  Industry:
Government  and Regulatory  Support :
Highlight and St rong suppor t  f rom governmen t  bod ies  and regu la to ry  agenc ies  g loba l l y  acce le ra t es  the  g rowth  o f  t he  pompe d isease  t r ea tment  indus t ry .  These  en t i t i es  
o f ten  exped i te  the  approva l  p rocesses  fo r  new therap ies  and  prov ide  f und ing  fo r  
Description research  and  deve lopmen t .  
Add i t i ona l l y ,  many  coun t r ies  have  imp lement ed  newborn  sc reen ing  programs  t ha t  
inc lude  t es t i ng  fo r  Pompe d isease,  lead ing  to  ea r l y  t rea t men t  in i t i a t i on  wh ich  can  
s i gn i f i can t l y  improve  pa t ien t  ou t comes .  Moreover ,  such  po l i c ies  encourage 
pharmaceu t i ca l  com pan ies  to  inves t  in  and  deve lop  new t rea tment s  and  ensure  
tha t  t hese  innova t ions  reach  t he  marke t  more  sw i f t l y ,  benef i t i ng  pa t ien t s  sooner .
Increased Awareness and Diagnosis:
Growing awareness of  pompe d isease among heal thcare  pro fess ionals  
and the publ ic  leads to ear l ie r  and more accurate  d iagnoses,  d i rect ly  
in f luenc ing the demand for  t reatment  so lu t ions.  Addi t iona l ly ,  educat ional  
campaigns and improved d iagnost ic  tools ,  such as genet ic  test ing,  are 
Report ident i fy ing more indiv iduals  who can benef i t  f rom t reatment  ear ly  in  the i r  d isease progress ion.  
As awareness increases,  so  does the engagement  of  medical  
Highlight and communi t ies  and governments in  address ing the needs of  th is  pat ient  
populat ion,  thus fur ther  expanding the growth of  the market  for  pompe 
Description disease t reatments .
Technological  Advancements:
The growth o f  the Pompe d isease t reatment  indust ry  i s  pr imar i ly  
in f luenced by breakthroughs in  b iotechnology  and genet ic  engineer ing.  
These advancements have led to  the deve lopment  of  more ef fect ive 
enzyme replacement  therap ies  (ERTs),  wh ich are cruc ia l  fo r  t reat ing 
Pompe disease by supp lement ing the def ic ient  enzyme in  pat ients .
Add i t iona l l y ,  p rogress  in  gene  the rapy  o f fe rs  the  po ten t ia l  fo r  l ong- te rm 
so lu t ions  by  address ing  the  gene t i c  roo t  causes  o f  the  d isease .  Moreove r ,  t h is  
con t inuous  evo lu t ion  in  t rea tment  approaches  enhances pa t ien t  ou tcomes and  
d r ives  indus t ry  expans ion  as  new and  improved  the rap ies  become ava i lab le  to  
a  b roader  pa t ien t  base .
Report 
Request  for  a  PDF sample  o f  th is  repor t :  
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Description
Report Description
Global  Pompe Disease Treatment Market  Trends:
The g loba l  Pompe d isease  t rea tment  marke t  i s  w i tness ing  s ign i f i can t  g rowth ,  p r imar i l y  d r iven  by  the  inc reas ing  
adop t ion  o f  enzyme  rep lacemen t  the rap ies  (ERTs) ,  wh ich  rema in  the  co rners tone  o f  t rea tment .  There  i s  a  g rowing  
emphas is  on  advanc ing  gene  the rapy  techn iques ,  p romis ing  po ten t ia l l y  cu ra t ive  bene f i t s ,  and  address ing  the  
under ly ing  gene t i c  causes  o f  Pompe d isease .  
Co l labo ra t ions  be tween  pharmaceu t ica l  compan ies  and  b io tech  f i rms  a re  a l so  in tens i fy ing ,  a imed  a t  acce le ra t ing  
the  deve lopment  and  d is t r ibu t ion  o f  i nnova t i ve  the rap ies .  Add i t iona l ly ,  the  marke t  s ign i f i can t l y  bene f i t s  f rom 
enhanced  inves tment  in  c l in i ca l  t r ia l s  t o  expand  the  e f f i cacy  and  app l ica t ion  range  o f  ex i s t i ng  t rea tments ,  
ensur ing  con t inua l  improvements  in  pa t ien t  ca re  and  ou tcomes and  thus  p rope l l i ng  marke t  g rowth .
Look ing  forward,  IMARC Group expects  the  market  to  reach US$ 1 .3  B i l l ion  by  2032 ,  exhib i t ing a  growth 
ra te  (CAGR) of  2 .58% dur ing 2024-2032 .
V iew Repor t  TOC,  F igures  and  Tables :  h t tps: / /www. imarcgroup.com/pompe-disease-t reatment -market
Treatment Insights:
• Enzyme Replacement Therapy (ERT)
• Substrate Reduct ion Therapy (SRT)
• Chaperone-Advanced Replacement Therapy 
(CART)
• Others
Report 
Route of Administration Insights:
Segmentation • Oral
• Intravenous
• Others
Distribution Channel Insights:
• Hospital  and Cl inics Pharmacies
• Retai l  Pharmacies
• Online Pharmacies
• Others
Indication Type Insights:
• Infant i le-Onset Pompe Disease ( IOPD)
• Classic Infant i le Form
• Non-Class ic  Infant i le Form
Report • Late-Onset  Pompe Disease (LOPD)
• Others
Segmentation
Regional Insights:
• North America
• Asia-Pacifi c
• Europe
• Lat in America
• Middle East  and Afr ica
• Amicus Therapeutics Inc.
• Audentes Therapeutics Inc.  (Astel las US 
Holding Inc.)
• Oxyrane UK Limited
Competitive 
• Sanofi  S.A.
Landscape • Spark Therapeutics Inc
with Key 
Players
How has the global Pompe disease treatment market 
performed so far and how will it perform in the coming 
years?
What are the drivers, restraints, and opportunities in 
the global Pompe disease treatment market?
Key 
What are the key regional markets?
Questions 
Answered in Which countries represent the most attractive Pompe 
disease treatment markets?
the Report
What is the breakup of the market based on the 
treatment?
What is the breakup of the market based on the route 
of administration?
What is the breakup of the market based on the 
distribution channel?
What is the breakup of the market based on the 
indication type?
Key 
What is the competitive structure of the global Pompe 
Questions disease treatment market?
Answered in 
Who are the key players/companies in the global Pompe 
the Report disease treatment 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  P o m p e  D i s e a s e  T r e a t m e n t  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 r e a t m e n t
    6 . 1     E n z y m e  R e p l a c e m e n t  T h e r a p y  ( E R T )
        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     S u b s t r a t e  R e d u c t i o n  T h e r a p y  ( S R T )
        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     C h a p e r o n e - A d v a n c e d  R e p l a c e m e n t  T h e r a p y  ( C A R T )
        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     O t h e r 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
7     M a r k e t  B r e a k u p  b y  R o u t e  o f  A d m i n i s t r a t i o n
    7 . 1     O r a l
        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     I n t r a v e n o u s
        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     O t h e r s
        7 . 3 . 1  M a r k e t  T r e n d s
Table of         7 . 3 . 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  &  C l i n i c s  P h a r m a c i e s  
Contents         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
    8 . 2     R e t a i l  P h a r m a c i e s  
        8 . 2 . 1  M a r k e t  T r e n d s
        8 . 2 . 2  M a r k e t  F o r e c a s t
    8 . 3     O n l i n e  P h a r m a c i e s  
        8 . 3 . 1  M a r k e t  T r e n d s
        8 . 3 . 2  M a r k e t  F o r e c a s t
    8 . 4     O t h e r s
        8 . 4 . 1  M a r k e t  T r e n d s
        8 . 4 . 2  M a r k e t  F o r e c a s t
9     M a r k e t  B r e a k u p  b y  I n d i c a t i o n  T y p e
    9 . 1     I n f a n t i l e - O n s e t  P o m p e  D i s e a s e  ( I O P D )
        9 . 1 . 1  M a r k e t  T r e n d s
        9 . 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 / p o m pe - d i s e a s e - t re a t m e n t - m a r ke t / t
o c
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