Pompe Disease Therapeutics Market Size, Share, Industry, Forecast and outlook (2024-2031)

Global Pompe Disease Therapeutics Market is segmented By Treatment Type (Drugs, Enzyme Replacement Therapy, Physical Therapy, Chaperone-Advanced Replacement Therapy), By Route of Administration(Oral, Parenteral), and By Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2024-2031

Pompe Disease Therapeutics Market Size

The global Pompe disease therapeutics market reached USD 1.3 billion in 2022 and is expected to witness lucrative growth by reaching up to USD 1.9 billion by 2031. The global Pompe disease therapeutics market is expected to exhibit a CAGR of 4.3% during the forecast period (2024-2031). Pompe disease is an inherited lysosomal condition induced by a lack of the enzyme acid alpha-glucosidase (GAA), which is supposed to result in the clinical presentations of Pompe disorder.

Pompe disease varies from a rapidly worsening infantile state with an influential effect on heart function, to a more slowly progressive, late-onset form predominantly impacting skeletal muscle and progressive respiratory involvement.

The increase in the number of cases, and the increasing market developments such as product approvals, mergers, acquisitions, expansions, and collaborations among other; rising clinical research activities for the Pompe disease therapeutics advancements and product approvals are among other factors expected to boost the global Pompe disease therapeutics market growth in the forecast period.

Pompe Disease Therapeutics Market Scope

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Pompe Disease Therapeutics Market Dynamics

Increasing Product Approvals Drive The Growth Of  The Pompe Disease Therapeutics Market

The increasing product approvals for the treatment of Pompe disease are expected to boost the global Pompe disease therapeutics market growth in the forecast period. For instance, in August 2021, Genzyme (Sanofi) Corporation, a subsidiary of Sanofi, obtained FDA authorization for its Nexviazyme (avalglucosidase alfa-ngpt) intravenous infusion in the treatment of patients with late-onset Pompe disorder aged one year and older. 

Approval is established on positive Phase 3 data indicating in key disease burden measures and establishing its safety profile. This application obtained fast track, priority review, and breakthrough therapy tags from the FDA. Nexviazyme was also specified as an orphan medication, which delivers financial inducements to support and encourage the development of drugs for rare conditions.

The Increase Awareness is Expected To Present The Global Pompe Disease Therapeutics Market With Growth Opportunities.

The increasing efforts to spread awareness about Pompe disease are presenting the global Pompe disease therapeutics market with lucrative growth opportunities. For instnace, the WORLD Symposium 2023, a 5-day conference was carried out in Orlando, Florida, in February 2023 and was a well-attended face-to-face conference. Considerable corporations with stakes in Pompe disorder were attending along with a satisfactory numeral of patient representatives throughout the event representing the UK, New Zealand, Germany, USA, Canada, and Japan.

Moreover, in April 2023 observed International Pompe Day and Pompe Support Network marked the day with an amazing turnout from the Pompe community at Pompe Spring Meeting, carried at the NeuroMusclar Centre in Winsford, Cheshire. Similarly, the Association for Glycogen Storage Disease (UK) Limited, marked International Pompe Day by focusing on raising awareness of the importance of movement.

The Presence of  Stringent Regulatory Bodies is Estimated To Hamper The Global Pompe Disease Therapeutics Market During The Forecast Period. 

The presence of stringent regulatory bodies for the approval of the newly developed Pompe disease therapeutics is hampering the global market growth in the forecast period. For instance, the Food and Drug Administration (FDA), a regulatory body is accountable for safeguarding general health by guaranteeing the safeness, effectiveness, and security of human and veterinary drugs, biological developments, medical devices, the nation's food supply, cosmetics, and products that radiate radiation. 

Moreover, the European Medicines Agency (EMA), an agency of the European Union in command of the evaluation and surveillance of pharmaceutical developments. The FDA and EMA are two major regulatory bodies among other country-specific regulatory bodies controlling the development and commercialization of pharmaceutical and medical devices.

Pompe Disease Therapeutics Market Segment Analysis

The global Pompe disease therapeutics market is segmented based on treatment type, route of administration, end-user, and region.

The Enzyme Replacement Treatment Type is Estimated to Dominate the Global Pompe Disease Therapeutics Market.

The enzyme replacement treatment type is estimated to hold about 35.6% of the global pompe therapeutics market owing to the increasing product authorization. For instnace, in November 2021 the Biologics License Application (BLA) for avalglucosidase alfa indicated for long-term enzyme replacement treatment for individuals with Pompe disease (acid-glucosidase deficiency) was authorized for regulatory inspection by the FDA. 

Moreover, in June 2022,  the European Commission presented a commerce license for Nexvia Dyme (alglucosidase alfa), an enzyme replacement therapy (ERT) for the long-term therapy of both late-onset and infantile-onset Pompe disorder, a rare, advanced, and debilitating muscle condition.

Source: DataM Intelligence Analysis (2023)

Global Pompe Disease Therapeutics Market Geographical Share

North America is estimated to hold Approximately 38.3% of the Market Share by 2030, Owing to The Increasing in Hospital-Associated Infections.

With the presence of government and non-government organizations participating in the fight against Pompe disease supporting the patients and spreading awareness, the presence of key market players in this region involved in research development, the region is estimated to dominate the global market holding about 38.3% of the global market by 2030. For instance, Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania (Penn) expanded their alliance with requests to seek cooperative investigation and development of novel gene treatments for lysosomal disorders (LDs) and 12 more rare disorders. 

The partnership has been extended from three to six agendas for rare genetic disorders and now includes Pompe disease, Fabry disease, CDKL5 deficiency disorder (CDD), Niemann-Pick Type C (NPC), Mucopolysaccharidosis Type IIIB (MPS IIIB), and as a next-generation schedule in Mucopolysaccharidosis Type IIIA (MPS IIIA), both even understood as an element of Sanfillipo Syndrome.

Source: DataM Intelligence Analysis (2023)

Pompe Disease Therapeutics Market Key Players

The major global players in the market include Genzyme (Sanofi), Amicus Therapeutics, Valerion Therapeutics, Audentes Therapeutics, Actus Therapeutics, BioMarin Pharmaceutical, EpiVax, Oxyrane, Sangamo BioSciences, Avrobio, Spark Therapeutics among others.

Russia-Ukraine Conflict Analysis

The Russia-Ukraine conflict is thought to hold a minimal influence on the global Pompe disease therapeutics market, as there is a low incidence of conditions and a lack of primary market players in this region. Though continuing to countenance invasions from Russian armies, Ukrainian clinics are forced by growing numbers of deaths and limited medical aid. There are shortages across all treatment types of medications. Again, the significance of the import and export of fundamental materials are expected to minimally impact the global Pompe Disease Therapeutics market growth in the forecast period.

Key Developments

• In August 2022, Aro Biotherapeutics’ ABX1100, an investigational Centyrin-siRNA conjugate to treat Pompe disease, obtained an orphan drug marker from the FDA.
• In January 2022, Hansa Biopharma AB, "Hansa", a frontier in enzyme technology for rare immunological diseases, formed a strategic agreement with Asklepios BioPharmaceutical, Inc. ("AskBio"), a wholly incorporated AAV gene therapy corporation and a fully held associate of Bayer AG committed to designing drugs that enhance the quality of life for individuals with genetic disorders. The partnership is aimed to assess the probable usefulness of imlifidase as a pre-treatment before the administration of AskBio's gene therapy in Pompe disease in a pre-clinical and clinical feasibility schedule for individuals with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus (AAV). 
• In August 2022, Nexviadyme, the new Enzyme Replacement Therapy (ERT) for Pompe, formulated by Sanofi, has been advised by NICE, the National Institute for Health and Care Excellence.

Why Purchase the Report?

• To visualize the global pompe disease therapeutics market segmentation based on treatment type, route of administration, end-user, and region, as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development. 
• Excel data sheet with numerous data points of pompe disease therapeutics market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available in Excel consisting of key products of all the major players.

The global pompe disease therapeutics market report would provide approximately 53 tables, 54 figures, and 195 Pages.

Target Audience 2024

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies