Pompe Disease Market Size (2024 - 2029)

Pompe Disease Market Analysis

The Pompe disease market is expected to register a CAGR of 4.5% over the forecast period.

The COVID-19 pandemic impacted market growth. It resulted in benign Pompe diseases in many individuals. For instance, according to the NCBI research study published in March 2022, in some Pompe disease patients, the COVID-19 infection could have a benign outcome. The same source also stated that individuals with Pompe disease were at a high risk of acquiring COVID-19 infection. Thus, the outbreak of the pandemic increased the incidence rate of Pompe disease, which significantly increased the demand for drugs and treatment for the disease and is expected to maintain the upward trend over the forecast period.

The market's growth is largely attributed to the launch of novel drugs and the continued uptake of long-term therapies. Pompe disease is a rare disease classified as early infantile or late-onset, majorly affecting skeletal muscle, heart defects, and breathing problems. For instance, according to the National Institute of Health publication from April 2022, Pompe disease affects 1 in 40,000 individuals in the United States. Moreover, according to the research study published in June 2021, pathogenic or likely pathogenic variants (PLPVs) were found in higher proportions in East Asian and African patients with classic infantile-onset Pompe disease. The same source also reported that in the general population, total carrier frequency (CF) and predicted genetic prevalence (pGP) were 1.3 percent (1 in 77) and 1:23,232, respectively. Thus, the rising prevalence of Pompe disease is anticipated to boost market growth over the forecast period.

The launch of novel drugs is anticipated to boost demand for the treatment of Pompe disease, which may further drive market growth. Moreover, increasing R&D expenditure globally will also promote working on novel therapeutics and the treatment of Pompe disease, as this disease mostly leads to death if not treated at an early stage. For instance, in February 2021, Genethon launched a gene therapy clinical trial for late-onset Pompe disease, led by the company Spark Therapeutics, using technologies developed at Genethon. Furthermore, increasing development in enzyme replacement therapies and gene therapy may also create a lucrative opportunity for the Pompe disease market over the forecast period, as this therapy is widely used to treat this disorder. Therefore, increasing product launches and research and development related to Pompe disease are anticipated to spur market growth over the forecast period.

However, high treatment costs associated with Pompe disease may restrain market growth over the forecast period.

Pompe Disease Industry Segmentation

As per the scope of the report, Pompe disease, also known as glycogen storage disease type II disorder, is an autosomal recessively inherited metabolic disorder caused by glycogen accumulation in the lysosome. Pompe disease is a rare (estimated at 1 in every 40,000 births), inherited, and often fatal condition that disables the heart and skeletal muscles. It is caused by mutations in a gene that makes an enzyme called acid alpha-glucosidase (GAA). The Pompe Disease Market is segmented by Route of Administration (Oral and Parenteral), Treatment Type (Substrate Reduction Therapy (SRT), Enzyme Replacement Therapy (ERT), and Others), End User (Hospitals, Ambulatory Surgery Centers, and Others), and Geography (North America, Europe, Asia Pacific, the Middle East and Africa, and South America). The report offers the value (in USD million) for the above segments. The market report also covers the estimated market sizes and trends for 17 countries across major regions globally. The report assigns a value of (USD million) to the aforementioned segments.