Gaucher Disease Market Type, Share, Size, Analysis, Trends, Demand and Outlook 2032

The market for Gaucher disease was estimated at USD 980.0 million in 2021, and it is anticipated to grow at a CAGR of 8.5% to reach around USD 1,800.0 million in 2029.

The rising prevalence of Gaucher disease, increasing awareness about Gaucher disease, and upsurge in the number of approved therapies for the treatment of Gaucher disease. Even it is the most prevalent lysosomal storage disorder, Gaucher disease is a rare disorder with an average prevalence of 1 in 100,000 individuals. However, some ethnic races such as Ashkenazi Jews are more inclined to this disorder. Ashkenazi Jews have an average disease prevalence of 1 in 100,000 individuals. However, the high cost related with the treatment of Gaucher’s disease may restrain the growth of this market during the forecast period.

Increasing prevalence of Gaucher disease backed by the growing rate of inheritance of the disease, accessory initiatives by governments round the world, and growing awareness concerning the disease is expected to be among the major factors to propel the expansion of the market. The increase of the Gaucher disease market will be additional ascribed to the rising investments and expenditure for the R&D activities to incessantly refer additional possible solutions for the treatment. Furthermore, increasing number of government initiatives to spread awareness and growing adoption rate of early diagnosis will result in the expansion of Gaucher disease market. On addition to this, increasing disposable income and rising need for better disease treatment options will boost the market’s growth rate.

The global market for Gaucher disease is growing owing due to the outbreak of the COVID-19. A huge demand for Gaucher disease has been created by the increase in patients around the world, the large number of vaccine trials and development, and the COVID-19 vaccination programmes. Hence, the COVID-19 pandemic increased the market size for Gaucher disease in 2021.

Disease Type Insights
The type 1 disease segment is expected to carry the largest market size by growing at a major CAGR over the forecast amount. The common symptoms of type 1 disease include embody spleen and liver enlargement, bone issues, and fatigue. the expansion of the phase will be related to the rising variety of individuals plagued by type-1 disease across the globe. As sourced from the National Gaucher Foundation, close to 95% of total cases area unit stricken by sort one Gaucher malady in western countries.

Some of the drugs received approvals recently for the Gaucher disease treatment. For instance, U.S. Food and Drug Administration (FDA) has approved multiple drugs in past several years for the treatment of disease. In 2012, Elelyso and in 2014, Cerdelga was permitted by FDA for type 1 Gaucher disease. In 2010, FDA approved Vpriv, an enzyme replacement therapy (ERT), for type 1 Gaucher disease. These drugs offer range of options based on their requests, in turn is anticipated to fuel the growth of the market.

Type 2 and 3 are rare types of this disease. Both these conditions are neuronopathic, causing CNS complications throughout the disease course. Infants with type 2 GD rarely survive over two years, due to lack of treatment and severity of disease progression.
Other rare types of the disease include perinatal lethal form and cardiac form. The perinatal lethal form is the most adverse type of the disease, leading to life-threatening complications in infants or fetuses. Cardiac form affects the heart, generally, causing calcification of heart valves.

Therapy Type Insights
The enzyme replacement therapy market is anticipated to grow at a substantial rate during the forecast period due to increasing cases of Gaucher disease worldwide. Around 10-20 cases per million are reported yearly in the U.S., however approximately 100 cases per million are reported in Europe and Asia.
However, ERT is predicted to lose shares in the coming years due to the high cost of therapy and troublesome dosing schedule. Furthermore, ERT is regularly used for type 1 and type 3 to address problems unrelated to CNS as replacement enzymes cannot enter blood brain barrier. The treatment landscape is observing a gradual shift towards substrate replacement therapy (SRT), controlled by Cerdelga and Zavesca.

Region Insights
North America is predicted to dominant the Gaucher disease market due to growing investigations for the development of new medication. In 2016, the human from National Institutes of Health researchers collaborated with National Institute of medical specialty Disorders and Stroke (NINDS) and therefore the National Center for Advancing Travel Sciences (NCATS). Researchers known a brand new molecule that shows promising approach for treatment of the rare Gaucher disease.

Europe is predicted to be the second largest marketplace for Gaucher malady treatment thanks to the initiative taken by European organizations to unfold awareness concerning Gaucher malady among the population. for example, in 2014, European Gaucher Alliance launched Gaucher Awareness Day reaching to unfold awareness concerning this rare malady among the European population.

Key Companies Insights
Key players are aiming on their technological partnerships, collaborations, and merger & acquisitions strategies to attain a competitive edge and expand their product portfolio & business footprint. The ongoing Covid-19 pandemic hindered the healthcare systems across the globe and drove market players to innovate products for unexploited opportunities.

Some of the key players operating in the global Gaucher disease market include:
• Genzyme Corporation (Sanofi)
• Pfizer, Inc.
• Shire Human Genetics Therapies, Inc.
• Actelion Pharmaceuticals Ltd. (Johnson & Johnson)
• AVROBIO, Inc.
• Bristol-Myers Squibb Company
• PerkinElmer Inc.
• Amicus Therapeutics, Inc.
• Moderna, Inc.
• Greenovation Biotech GmbH
• Biomarin
• Takeda Pharmaceutical Company Limited
• AstraZeneca PLC
• Eli Lilly and Company
• Other players
There are several drugs in pipeline of companies such as Lixte Biotechnology Holdings Inc., JCR Pharmaceuticals Co Ltd., Pharming Group NV, and Orphazyme ApS

Some of the Recent Developments:
• In 2017, Janssen acquired Actelion to add complementary therapeutics and promising late-stage pipeline candidates to its portfolio.
• In 2015, Protalix sold worldwide rights of Elelyso to Pfizer.
• In 2012, Protalix entered into a licensing deal with Pfizer in 2009 for clinical development and commercialization of Elelyso.

Segments 
By Disease Type
• Type 1 Gaucher disease
• Type 2 Gaucher disease
• Type 3 Gaucher disease

By Therapy Type
• Enzyme Replacement Therapy (ERT)
• Substrate Reduction Therapy (SRT)

By End Users
• Hospitals
• Specialty Clinics
• Homecare
• Others

By Distribution Channel
• Hospitals
• Retail Pharmacies
• Online Pharmacies

By Geography
• North America
o U.S.
o Canada
o Mexico
• Europe
o U.K.
o Germany
o France
o Italy
o Spain
o Russia
• Asia-Pacific
o Japan
o China
o India
o Australia
o South Korea
o ASEAN
• Latin America
o Brazil
o Argentina
o Colombia
• MEA
o South Africa
o Saudi Arabia
o UAE
o Egypt