Global Pompe Disease Therapeutics Market - 2023-2030

Market Overview

The global Pompe disease therapeutics market reached US$ 1.3 billion in 2022 and is expected to witness lucrative growth by reaching up to US$ 1.9 billion by 2030. The global Pompe disease therapeutics market is expected to exhibit a CAGR of 4.3% during the forecast period (2023-2030).

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.

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 June 2023, Amicus Therapeutics, a patient-dedicated multinational biotechnology corporation concentrated on designing and commercializing novel therapies for rare conditions, obtained the European Commission (EC) authorization for its Opfolda (miglustat) 65mg capsules, an enzyme stabilizer of cipaglucosidase alfa, a long-term enzyme replacement therapy for adults with late-onset Pompe disease.

Furthermore, Amicus intends to instantly launch Pombiliti (cipaglucosidase alfa) was earlier authorized by the EC in March 2023 in combination with Opfolda, a two-component therapy that consists of cipaglucosidase alfa and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood in Germany and is beginning reimbursement strategies with healthcare authorities in other European countries.

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 instance, in April 2023, the Pompe Support Network marked International Pompe Day and honored the day with a theme of "Every Move Counts", an exceptional turnout from the Pompe Society at the Pompe Spring Meeting was observed at the event conducted at the NeuroMusclar Centre in Winsford, Cheshire.

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) and the European Medicines Agency (EMA) are two major regulatory bodies among other country-specific regulatory bodies controlling the development and commercialization of pharmaceutical and medical devices.

COVID-19 Impact Analysis

The financial health of companies across all industries has been impacted by the COVID-19 pandemic and lockdown in numerous nations throughout the world. Therefore, for the period of the COVID-19 public health emergency, the U.S. Food and Drug Administration (FDA) issued guidelines that include general considerations to aid sponsors and researchers, ensuring the safety of trial participants, adhering to good clinical practice (GCP), and minimizing risks to trial integrity.

Russia-Ukraine War Impact 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.

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 27% of the global pompe therapeutics market owing to the increasing advancements in the therapy. For instance, in November 2022, the specialists at Duke Health were in a multi-national crew concerned with treating a fetus for infantile-onset Pompe disease utilizing an enzyme replacement therapy, a first in the world. The case, which was documented online on November 9 in the New England Journal of Medicine, emphasizes the possibility of introducing treatment for Pompe disease before birth.

Geographical Analysis

Europe is Estimated to Hold 27.4% of the Market Share Owing to the Presence of Key Market Players in this Region.

Owing to the presence of key market players involved in distinct market tactics holding a majority of the market and the presence of distinct organizations working to raise funding and spread awareness about conditions, the European Pompe disease therapeutics market is estimated to hold about 29% of the global market share.

For instance, Sanofi, a French global pharmaceutical and healthcare corporation, and Maze Therapeutics, an operator of a biotechnology firm utilized to concentrate on deciphering genetical understandings into new medicines formed a contract for the sole global consent of Maze's glycogen synthase 1 (GYS1) program and oral substrate reduction therapy, MZE001, to treat Pompe disease.

Competitive Landscape

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.

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 2023

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