What is the current size and annual growth rate of the pombiliti market?
The pombiliti market size has XX (HCAGR) in recent years. It will grow from $XX million in 2024 to $XX million in 2025 at a compound annual growth rate (CAGR) of XX%. The growth in the historic period can be attributed to rising prevalence of pompe disease, growth of enzyme replacement therapies globally, expansion of clinical trials, rising healthcare expenditure, and rising incidence of metabolic disorders.
The pombiliti market size is expected to see XX (FCAGR) in the next few years. It will grow to $XX million in 2029 at a compound annual growth rate (CAGR) of XX%. The growth in the forecast period can be attributed to growth in demand for personalized medicine, rising incidence of chronic diseases, increasing prevalence of lifestyle-related diseases, rising demand for comprehensive patient support programs, and rising incidence of comorbid conditions. Major trends in the forecast period include advancements in gene therapy, advancements in drug delivery systems, advancements in biomarker discovery, advancements in clinical trial designs, and development of companion diagnostics.
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Which major factors have contributed to the expansion of the pombiliti market?
Rising prevalence of pompe disease is expected to propel the growth of the pombiliti market going forward. Pompe disease refers to a rare genetic disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), leading to the accumulation of glycogen in muscle cells, which results in progressive muscle weakness, respiratory difficulties, and impaired heart function. The prevalence of Pompe disease is increasing due to advancements in diagnostic tools, expanded newborn screening programs, and improved awareness, leading to earlier detection and higher reported cases globally. Pombiliti helps treat Pompe disease by providing a recombinant human acid alpha-glucosidase enzyme replacement therapy that improves muscle function and reduces glycogen buildup, addressing the underlying cause of the disease and enhancing patients’ overall quality of life. For instance, in September 2023, according to a report published by the Egton Medical Information Systems Limited, a UK-based private limited company, pompe’s disease has an estimated prevalence of 1 in 40,000 among African Americans, 1 in 50,000 among Chinese individuals, 1 in 40,000 among the Dutch population, and 1 in 146,000 among Australians. While the commonly cited frequency of the disease is 1 in 40,000 live births, newborn screening has indicated that the actual prevalence may be significantly higher. Therefore, the rising prevalence of Pompe disease is driving the growth of the pombiliti market.
How is the pombiliti market segmented?
The pombiliti market covered in this report is segmented –
1) By Indication: Late-Onset Pompe disease
2) By Administration Route: Intravenous Infusion
3) By Distribution Channel: Direct Sales; Pharmaceutical Wholesalers; Online Pharmacies
4) By End-User: Hospitals; Clinics
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Who are the top competitors in the pombiliti market?
Major companies operating in the pombiliti market are Amicus Therapeutics Inc.
Which key trends are expected to influence the pombiliti market in the coming years?
The key trend in the Pombiliti market is a strong emphasis on developing innovative therapies to enhance treatment efficacy for patients with Pompe disease. The innovative therapy pairs pombiliti, an enzyme replacement treatment, with opfolda, an oral stabilizer, to significantly improve patient outcomes. For instance, in September 2023, Amicus Therapeutics Inc., a US-based biotechnology company, announced that the US Food and Drug Administration (FDA) approved the combination therapy of Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) in 65 mg capsules. The combination therapy of Pombiliti and Opfolda represents a significant advancement in the treatment of late-onset Pompe disease. Pombiliti, an enzyme replacement therapy enriched with bis-M6P, enhances enzyme uptake by muscle cells, while Opfolda functions as an oral enzyme stabilizer that reduces the inactivation of Pombiliti in the bloodstream.
Which regional trends are influencing the pombiliti market, and which area dominates the industry?
North America was the largest region in the pombiliti market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the pombiliti market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
What Does The Pombiliti Market Report 2025 Offer?
The pombiliti market research report from The Business Research Company offers global market size, growth rate, regional shares, competitor analysis, detailed segments, trends, and opportunities.
Pombiliti, also known by its generic name cipaglucosidase alfa, is an enzyme replacement therapy (ERT) developed for the treatment of late-onset Pompe disease (LOPD). Pompe disease is a rare genetic disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), which leads to the buildup of glycogen in muscle cells, resulting in progressive muscle weakness and respiratory issues.
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