The UK Gaucher Disease Therapeutics market size stood at around USD XX billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
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. It is the most common lysosomal storage disorder (LSD). Gaucher disease is caused by mutations of a single gene called GBA, which leads to very low levels of glucocerebrosidase enzyme leading to low degradation of glucocerebroside.
The symptoms and physical findings associated with Gaucher disease vary greatly from patient to patient. Some individuals are asymptomatic; develop few or no symptoms while others may have serious complications include skeletal abnormalities, blood disorders, and abdominal complaints. Three separate forms of Gaucher disease have been identified and are distinguished by the absence of, or the presence and extent of, neurological complications. All three forms of Gaucher disease are inherited in an autosomal recessive pattern.
Among these, Gaucher disease type 1 is the most common type. Over 90% of people affected have type 1 Gaucher disease. The overall frequency of all types of Gaucher disease is approximately 1 in 50,000 to 1 in 100,000 live births. Clinical experts estimate that there are between 350 to 400 patients with Gaucher disease in the UK; out of these 84% reside in England and 86% over 18 years of age. Each pregnant female has a 1 in 4 chance that the baby will be born with the disease. The age of onset for Gaucher disease type 2 is during early infancy. The age of onset of Gaucher disease type 3 varies, but the disorder generally begins during childhood or adolescence. The mean age at diagnosis is 24 years and at symptom onset is 11 years.
Market Growth Drivers
The Gaucher disease treatment market is likely to exhibit modest growth during the forecast period. Rising incidence of the disease and increasing efforts for the identification of more Gaucher disease patients are among the primary growth stimulants for the market.
An increase in the number of drug approvals for the management of disease and high investments by drug companies in research and development activities to develop novel therapies for its treatment are projected to shape the future of the Gaucher disease treatment market during the forecast period.
Whereas, lack of awareness about Gaucher disease among people is expected to restrain the market growth during the forecast period. Additionally, the lack of drugs in the late-stage pipeline and the rising cost of treatment are limiting the Gaucher disease treatment market from realizing its utmost potential.