Gaucher disease – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

 

Indication name: Gaucher disease

Gaucher disease – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy, Japan & China).

Gaucher disease is a rare, inherited metabolic disorder in which deficiency of the enzyme glucocerebrosidase results in the accumulation of harmful quantities of certain fats (lipids), specifically the glycolipid glucocerebroside, throughout the body especially within the bone marrow, spleen and liver. Gaucher disease is caused by changes (mutations) in the GBA gene.

Gaucher disease divided into 3 different types based on the presence or absence of early-onset brain involvement, including:

Gaucher disease type 1 is also known as non-neuronopathic, experience easy bruising due to low levels of blood clotting cells known as platelets (thrombocytopenia), chronic fatigue due to low levels of circulating red blood cells (anemia), and an abnormally enlarged liver and/or spleen (hepatosplenomegaly).

Gaucher disease type 2, also known as acute neuronopathic Gaucher disease, occurs in newborns and infants and is characterized by neurological complications due to the abnormal accumulation of glucocerebroside in the brain.

Gaucher disease type 3, also known as chronic neuronopathic Gaucher disease, occurs during the first decade of life. Associated neurological complications include mental deterioration; an inability to coordinate voluntary movements (ataxia); and brief, shock-like muscle spasms of the arms, legs or entire body (myoclonic seizures).

Epidemiology-

Gaucher disease can affect anyone, occurring in up to 1 in 40,000 live births in the general population. Literature suggest it is more common among Jews of Ashkenazi (Eastern European) descent, occurring in approximately 1 in 450 within this population. As many as 1 in 10 may carry the mutated gene responsible for Gaucher disease.

Competitive landscape of Gaucher disease includes country specific approved as well as pipeline therapies. Any asset/ product specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

KOLs insights of Gaucher disease across 8 MM market from center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm and Unmet needs.

Gaucher disease Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which Data Inputs with sourcing, Market Event and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden and pricing scenario, Summary and Insights.

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S. No     Asset     Company           Stage

1            PR001   Prevail Therapeutics      Phase 2

2            GA-GCB (velaglucerase alfa)        Shire     Phase 2

3            AVR-RD-02         AvroBio Phase 2

4            venglustat (GZ402671)   Genzyme, a Sanofi Company      Phase 3

5            Arimoclomol     Orphazyme        Phase 2

6            Abcertin             ISU Abxis Co., Ltd.           Phase 1

7            Miglustat            Actelion              Phase 3

8            afegostat tartrate           Amicus Therapeutics      Phase 2

9            PRX-112              Protalix Phase 2