Pompe disease is a disorder caused by the buildup of a complex sugar called glycogen in the body's cells. It is genetically inherited and the accumulation of glycogen in certain organs as well as tissues, especially muscles, impairs their ability to function normally. Pompe diseases affects approximately one individual in 40,000 in the U.S.; however, the incidence varies among different ethnic groups.
Increasing research activities preformed to find an effective treatment for the disease and the growth in the special regulatory drug designations for orphan drugs drive the market. However, inflated costs associated with the Pompe disease therapy restrain the market growth. Moreover, emerging immunotherapeutic approaches for Pompe disease offer lucrative opportunities for the expansion of the market.
The market is segmented on the basis of therapy type, molecule type, route of administration, dosage forms, and region. Based on therapy type, the market is divided into enzyme replacement therapy and substrate reduction therapy. Based on the molecule type, the market is classified into biologics and small molecules. Based on route of administration, the market is bifurcated into oral and parenteral. Based on the dosage forms, the market is categorized into solid and liquid. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.
Key players that operate in the market are Amicus Therapeutics, BioMarin Pharmaceutical, Genzyme, Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.
- The study provides an in-depth analysis of the global Pompe disease market with current trends and future estimations to elucidate imminent investment pockets.
- It presents a quantitative analysis from 2016 to 2023 to enable stakeholders to capitalize on prevailing market opportunities.
- Key players are profiled and their strategies are thoroughly analyzed, which predicts the competitive outlook of the market.
- By region, the market is analyzed across North America, Europe, Asia-Pacific, and LAMEA.
Pompe Disease Market Key Segments:
By Therapy Type
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
By Molecule Type
- Small Molecules
By Route of Administration
By Dosage Forms
- North America
- Rest of Europe
- Rest of Asia-Pacific
- Saudi Arabia
- South Africa
- Rest of LAMEA