Global Pompe Disease Market to 2030 - Insights, Epidemiology and Forecast - ResearchAndMarkets.com
The "Pompe Disease - Market Insights, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.
The "Pompe Disease - Market Insights, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.
This 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.
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 -1, 4- and -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.
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 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 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.
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, 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 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.
Companies Mentioned
- Genzyme
- Sanofi
- Amicus Therapeutics
- Actus Therapeutics
- Valerion Therapeutics
- Astellas Therapeutics
- Roche
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Lacerta Therapeutics
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