Pompe Disease Market Report 2030 | Pompe Disease Market Scenario 2030

1. Pompe Disease Market DelveInsight’s "Pompe Disease (PD) - Market Insights, Epidemiology and Market Forecast- 2030" report delivers an in-depth understanding of the disease, historical &forecasted epidemiology as well as the market trends of PD in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom) and Japan. The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Pompe Disease (PD) from 2017 to 2030 segmented by seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market. Geography Covered    The United States EU5 (Germany, France, Italy, Spain and the United Kingdom) Japan Study Period: 2017–2030 View report: https://www.delveinsight.com/sample-request/pompe- disease-market Pompe Disease (PD) - Disease Understanding and Treatment Algorithm Pompe disease (PD), also known as glycogen storage disease type II (GSDII) or “acid maltase deficiency”, is caused by the absence or deficiency of acid alpha-glucosidase (GAA), a lysosomal enzyme that is responsible for the cleavage of the α-1, 4- and α-1, 6-glycosidic bonds of glycogen to glucose. The disease is caused pathogenic variations in the acid alpha-glucosidase (GAA) gene. Close to 500 different GAA gene variations have been identified in families with this disorder.

2. The treatment of Pompe disease is disease-specific, symptomatic, and supportive. Treatment requires the coordinated efforts of a team of specialists with expertise in treating neuromuscular disorders. Pediatricians or internists, neurologists, orthopedists, cardiologists, dieticians, and other healthcare professionals may need to systematically and comprehensively plan an affect child’s treatment. Enzyme replacement therapy is an approved treatment for all patients with Pompe disease. It involves the intravenous administration of recombinant human acid α-glucosidase. This treatment, manufactured by Genzyme, a Sanofi Corporation. Pompe Disease Diagnosis The diagnosis of Pompe disease often poses a diagnostic dilemma due to the rarity of the condition and the relatively nonspecific nature of the phenotypic features that may only lead to suspicion of Pompe disease. A chest x-ray and electrocardiogram (ECG) are valuable screening tests in the diagnostic algorithm for infantile Pompe disease and an echocardiogram is a valuable next step. Chest x-ray shows massive cardiomegaly. ECG shows a short PR interval as well as very tall QRS complexes. Among patients presenting with cardiomyopathy, an electromyogram (EMG) can be useful to document presymptomatic myopathy. Blood tests should include a serum creatine kinase (CK) examination. CK elevation is a sensitive although very nonspecific marker for Pompe disease. Pompe Disease Treatment It covers the details of conventional and current medical therapies available in the Pompe Disease market for the treatment of the condition. It also provides the country-wise treatment guidelines and algorithm across the United States, Europe and Japan. The DelveInsight Pompe Disease market report gives a thorough understanding of PD by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides PD treatment algorithms and treatment guidelines in the US, Europe, and Japan. Pompe Disease Epidemiology The Pompe Disease (PD) epidemiology division provide the insights about historical and current patient pool and forecasted trend for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken. Key Findings The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total live birth cases of PD, Total Adult Prevalent cases of PD, Total Prevalent cases of PD, Comorbidities Prevalence of PD by Onset Types, Incidence of IOPD based on clinical phenotypes) scenario of Pompe Disease (PD) in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017–2030. Request for sample pages: https://www.delveinsight.com/sample- request/pompe-disease-market Pompe Disease Drug Chapters This segment of the Pompe Disease report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the clinical trial details,

3. expressive pharmacological action, agreements and collaborations, approval and patent details and the latest news and press releases. The Pompe Disease (PD) drugs market is dominated by the use of Enzyme Replacement Therapies (ERT) along with the use of supportive therapies for the management of PD. Most patients require supportive therapy to address the symptoms of Pompe disease, which include respiratory and cardiac problems, physical disability, and difficulty swallowing. Although historically, supportive care had been the mainstay of treatment (thereby generating a huge amount of revenue), but the approval and subsequent launch of ERT in 2006 after decades of research, helped turn the treatment landscape upside down, prompting a new era in the treatment of this disease. This was the first disease-specific treatment for an inherited muscular disorder consisting of the intravenous administration of recombinant human GAA (Myozyme / Lumizyme). Pompe Disease Marketed Drugs Lumizyme/ Myozyme (alglucosidase alfa): Sanofi Genzyme LUMIZYME (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency). It is an enzyme replacement therapy produced by recombinant DNA technology. It provides an exogenous source of acid α glucosidase (GAA), an essential lysosomal enzyme that is deficient or absent in patients with Pompe disease, leading to intralysosomal glycogen accumulation. Products detail in the report… Pompe Disease Emerging Drugs AT-GAA: Amicus Therapeutics AT-GAA is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In Feb 2019, the US Food and Drug Administration (FDA) granted Amicus a Breakthrough Therapy Designation (“BTD”) to AT-GAA for the treatment of late-onset Pompe disease. Products detail in the report… Pompe Disease Market Outlook The Pompe Disease market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology. This segment gives a through detail of market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight. According to DelveInsight, the market of Pompe Disease in 7MM is expected to undergo a major positive shift during the course of the study period (2017–2030).Among the 7MM, the United States accounts for the largest market size of PD, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.

4. Key Findings This section includes a glimpse of the Pompe Disease market in 7MM. The United States Market Outlook This section provides the total Pompe Disease market size and market size by therapies in the United States. EU-5 Countries: Market Outlook The total Pompe Disease market size and market size by therapies in Germany, France, Italy, Spain and the United Kingdom is provided in this section. Japan Market Outlook The total Pompe Disease market size and market size by therapies in Japan is also mentioned. Download request/pompe-disease-market sample pages: https://www.delveinsight.com/sample-