Global Gaucher Disease Market 2020 and Forecast 2021-2026

                                          
Gaucher disease is a genetic disorder where fat-laden Gaucher cells build up in areas like the spleen, liver and bone marrow. A person will get Gaucher Disease if both parents are carriers of the disease. In this inherited disorder where the body does not make enough glucocerebrosidase (GCase), an enzyme that breaks down fatty chemicals in the body. GCase is often mistakenly referred to as GBA, which is actually the gene that affects GCase production.

Type 1: Gaucher disease type 1 makes up the majority of cases in western countries. Symptoms and signs can include bone and organ problems, but brain development is normal. This form of the disease is treatable. Find out more about type 1.

Type 2: Also called acute infantile neuronopathic Gaucher disease, Gaucher disease type 2 has an onset of central nervous system problems that are typically fatal within 2 years. The central nervous system problems of type 2 are not treatable.

Type 3: Gaucher disease type 3 is the most common variant of the disease worldwide. Symptoms and signs can include bone and organ problems, as well as neurological effects such as seizures. It typically has a later onset compared with type 2, and patients may survive into adulthood.

Enzyme replacement therapy (ERT) and cost:


Enzyme replacement therapy (ERT) balances low levels of GCase in patients with Gaucher disease so their bodies can break down glucocerebroside. (This compensates for the missing enzyme, which is why the therapy is called enzyme replacement.) ERT involves receiving intravenous (IV) infusions about every 2 weeks, either at an infusion center or at home. In the U.S. ERT can be very expensive, totaling up to USD $200,000 or more each year. Appropriate insurance may cover most of this cost, and resources are available.

Substrate reduction therapy (SRT)

Substrate reduction therapy (SRT) is a newer treatment that works differently than ERT. SRT is an oral medication that decreases the amount of glucocerebroside that the body makes, reducing excess buildup. Zavesca cost around $27000 for 90 capsules while generic Zavesca cost around $20,000 in the United States without insurance. With insurance, they are available from $50 to $26200. While Cerdelga costs upwards of $40000 for 90 capsules without insurance. Currently, there are no generics available for Cerdelga.

In 2020, the global Gaucher Disease market size was XX million US$ and it is expected to reach XX million US$ by the end of 2026, with a CAGR of during 2021-2026.

This report focuses on the global Gaucher Disease status, future forecast, growth opportunity, key market, and key players. The study objectives are to present the Gaucher Disease development in the United States, Europe, Asia Pacific, and the Middle East and Africa.

The key players covered in this study

 	
Sanofi
 	
Shire
 	
Actelion Pharma
 	
Takeda Pharmaceuticals America
 	
CoTherix, Inc.
 	
ANI Pharmaceuticals, Inc
 	
Edenbridge Pharmaceuticals LLC.
 	
Pfizer
 	
ISU Pharm

Market segment by Type, the product can be split into

 	
Enzyme Replacement Therapy
 	
Substrate Reduction Therapy

Market segment by Application, split into

 	
Non-Neuronopathic Gaucher Disease
 	
Neuronopathic Gaucher Disease

Market segment by Regions/Countries, this report covers

 	
United States
 	
Europe
 	
Asia-Pacific
 	
Central & South America
 	
Middle East Asia and Africa

The study objectives of this report are:

 	
To analyze global Gaucher Disease status, future forecast, growth opportunity, key market and key players.
 	
To strategically profile the key players and comprehensively analyze their development plan and strategies.
 	
To define, describe, and forecast the market by product type, market, and key regions.