ID: MRFR/HC/6761-CR | January 2020 | Region: Global | 123 pages
Pompe disease is a rare and fatal neurological disorder that causes the degeneration of the brain cells. This leads to tremors, seizures, and cognitive impairment. The Pompe disease treatment market has been growing steadily over the past few years. The prevalence of Pompe disease in the U.S is estimated at about 1 in 200,000 people with an annual incidence rate of about 1 case per 800,000 people. Pompe disease affects many different parts of individuals’ lives including their professional lives as it negatively impacts their career prospects.
Pompe Disease Treatment Market consists of both direct-to-consumer (DTC) and healthcare providers. DTC market includes various treatment options like stem cell transplant, bone marrow transplantation, chemotherapy, immunotherapy etc. Healthcare providers include academic medical centres and private practices. It is expected that the total spend on Pompe Disease Treatment Market is expected to register a CAGR of 3.0% during the forecast period of 2019 to 2030 and was valued at USD 1,181.6 Million in 2018.
COVID-19 Analysis of Pompe Disease Treatment Market
The pandemic caused by COVID-19 has affected the supply chain and the production of medical products for Pompe disease. The market may face disruption due to a lack of attendance of human labour at work and in factories. However, there is a silver lining. Just like any other market, advanced technology has taken place instead of human labour. Though it may need human interventions from time to time, it is indeed scarce.
The government of various nations are running vaccine drives. The initial months of the COVID-19 pandemic saw a decline in production of the market. However, the Pompe Disease Treatment Market has paced up production.
Pompe Disease Treatment Market Share, by Type, 2018 (%)
Source: MRFR Analysis
The Pompe Disease Treatment Market is driven by several factors such as increasing awareness, legislation, reimbursement policies of health insurance companies, and technological advancements. Pompe disease treatment market has been on the rise globally due to increasing awareness about the disease as well as its potential impact on society. The success of treatments is also strongly influenced by changes in legislation such as laws concerning reimbursement for Pompe Disease Treatment by health insurance companies.
The global healthcare industry continues to adopt new technologies every day which would positively influence Pompe Disease Treatment market size in future years. The treatment market for Pompe disease is growing due to the increase in the prevalence of the disease. The drivers for this market are increasing incidence rates, the presence of Pompe disease in developing countries, and the lack of effective treatments for this disease.
While the Pompe disease treatment market is expected to grow at a high rate in the coming years, it faces several restraints such as lack of well-defined efficacy, high cost, and drug regulatory issues.
Pompe disease is not curable but many therapies can help manage its symptoms. The main therapy includes enzyme replacement therapy or ERET which provide an alternative to the current therapies for the treatment of Pompe disease.
In the last few years, awareness about Pompe disease has been increasing in the global audience. This is because of the increasing number of people diagnosed with this hereditary disorder.
Pompe disease is a disorder that affects a person’s muscle and brain function. Patients with this disorder experience muscle spasms and eventually lose control over their bodily functions resulting in death. However, there are many treatment options available for patients to help manage the symptoms of the condition. The market size for these treatments is expected to grow USD 4 billion by 2025.
The growing prevalence of Pompe disease provides opportunities for treatment providers who have developed new products and services focused on improving patient outcomes through medication, medical devices, pharmaceuticals, surgical procedures, rehabilitation therapies and other interventions.
The treatment for this condition is expensive, and only about 1/3 of patients who receive it survive beyond five years. There’s a high degree of unmet medical need for Pompe disease treatment, which means that the market for this type of therapy is ripe for disruption.
Many challenges are facing the Pompe Disease Treatment Market, but one of the most significant ones is low adoption rates because people with this condition are not able to afford treatment.
Value Chain Analysis
As per the value chain analysis of the Pompe Disease Treatment Market, there are different stakeholders involved in the market such as pharmaceutical companies, healthcare providers, medical professionals and patients. The key players in this niche are Pfizer Inc., Hospira Inc., AstraZeneca PLC and Merck & Co., Inc.
Some companies are now developing new methods to treat the disease, such as the use of antibiotics combined with gene therapies. However, these methods are still not easy for people with low incomes to afford. Other diseases require treatment but there aren't many solutions available for them either.
The value chain can be divided into three parts: basic research, innovation, and commercialization. Basic research focuses on scientific discovery, innovation creates novel technologies, and commercialization creates marketable products ready for consumers. The value chain also includes healthcare providers.
Pompe Disease Treatment Market Share, by Region, 2018 (%)
Source: MRFR Analysis
Pompe Disease Treatment Market Segment Overview
There are five types of Pompe Disease Treatment Market:
Pompe disease treatment market is expected to reach USD 1.5 billion by 2025 with the presence of global key players like Bayer AG, BMS, and CSL. The regional Pompe Disease Treatment market is analyzed through the use of a bottom-up approach. North America is projected to dominate the global Pompe Disease Treatment market in terms of revenue share by 2025.
The regional analysis of Pompe Disease Treatment Market report includes key insights on top markets including North America, Europe, Asia Pacific & the Rest of the World (RoW), The United States holds the largest share of the market while Italy has the highest revenue share among other countries in Europe. North America has been the largest regional market in 2015 and accounted for more than 50% of the overall global Pompe Disease Treatment Market share. With its high disposable income, North America is expected to maintain its dominance.
With the increase in the number of cases of Pompe Disease, treatment and prevention have become a priority for both national and global health organizations.
The US National Institutes of Health (NIH) conducted a study to find out which is the best drug for Pompe Disease treatment. The study revealed that small molecule drugs like pegylated liposomal doxorubicin (DOXO) and pomalidomide may be more effective than other drugs like cilengitide and panobinostat.
However, the study also revealed that these drugs may not be affordable enough for most patients with Pompe Disease. Hence, there is a need to explore alternative therapies that can reduce the cost of treatment and prevention.
The Pompe Disease treatment market is growing exponentially. With this increase in demand for new treatments, the competition is fierce among the pharmaceutical industry players. The challenge for this company will be to stay ahead of its competitors.
The Pompe Disease Treatment Market report traces different sectors of the market like drivers, opportunities, restraints and others. Pompe disease is rare and expensive to treat. It is also a non-curable disease. Developments in the treatments of the disease are still going on.
This disease has an average life expectancy of three to five years. It can be fatal if left untreated. However, there is hope for this deadly disease. Pompe Disease Treatment uses gene therapy to stop the immune system from attacking neurons and stop the progression of the disease.
Pompe disease treatment includes hospitalization followed by intravenous injections of drugs which help patients to maintain cognitive function until their bodies can no longer produce enough enzymes to break down toxic metabolites.
|Market Size||2030: Significant Value|
|Forecast Units||Value (USD Million)|
|Report Coverage||Revenue Forecast, Competitive Landscape, Growth Factors, and Trends|
|Segments Covered||Type, Therapy, End-User and Region|
|Geographies Covered||North America, Europe, Asia-Pacific, and Rest of the World (RoW)|
|Key Vendors||Amicus Therapeutics, Inc (US) Sanofi (France), Audentes Therapeutics (US), Oxyrane (UK), Valerion Therapeutics (US), AVROBIO, Inc. (US), CENTOGENE AG (UK).|
|Key Market Opportunities||The rising prevalence of Pompe disease is creating opportunities for manufacturers to find new solutions for the diagnosis and treatment|
|Key Market Drivers||
Frequently Asked Questions (FAQ) :
Pompe disease treatment market was valued at close to USD 1.2 billion in 2018.
Global market is expected to exhibit a strong 3% CAGR over the forecast period from 2019 to 2030.
Increasing R&D initiatives by drug manufacturers are the major driver for the pompe disease treatment market.
The Americas hold the dominant share in the global market, followed by Europe.
Leading players in the market include Amicus Therapeutics, Valerion, and Sanofi, among others.