The market for Gaucher disease, a rare inherited metabolic disorder characterized by hepatosplenomegaly, bone disease, and anemia, is set to experience a compound annual growth rate (CAGR) of 0.79% across the seven major markets (7MM*) from $1.2 billion in 2022 to $1.3 billion in 2032, forecasts GlobalData, a leading data and analytics company.

GlobalData’s latest report, “Gaucher Disease: Opportunity Assessment and Forecast”, reveals improved testing approaches, increased diagnosis, and the launch of venglustat malate will be the main drivers of steady market growth over the forecast period.

Sulayman Patel, MSci, Pharma Analyst at GlobalData, comments: “Improvements in diagnostic approaches, particularly by increased inclusion of Gaucher disease in screening panels, as well as increased patient numbers in line with population growth, will increase market size in the next decade. Additionally, market growth will be further enhanced by venglustat malate, which GlobalData anticipates will launch in the US and 5EU* markets during the forecast period.”

The Gaucher disease market has been historically dominated by enzyme replacement therapy (ERT), however, GlobalData forecasts that substrate reduction therapy (SRT) will increase market share in the next 10 years, as younger patients increasingly switch from ERT to SRT. Key opinion leaders (KOLs) interviewed by GlobalData stated that older patients are often reluctant to switch to SRT, based on familiarity provided by receiving ERT.

Patel continues: “The approval of venglustat malate is predicted to catalyze the increased patient share of SRT within the Gaucher disease landscape. As the first brain-penetrant SRT, venglustat malate holds the potential to provide relief for neuropathic symptoms. In turn, this will address the most significant unmet need highlighted by KOLs, the lack of effective therapies for neuropathic symptoms.”

GlobalData has identified several barriers to the slow expansion of the market during the forecast period. Primarily, the Gaucher disease market is crowded with expensive ERTs and SRTs, where biosimilars face difficult market penetration due to strict regulations, resulting in reduced market growth. This constraint on the market’s growth momentum is intensified by strong physician and patient trust in existing therapies, hindering the growth of prospective players entering the market.

Patel concludes: “Despite the predicted increase in SRT patient share, GlobalData predicts ERT to continue to be the most profitable drug class in the Gaucher disease space by 2032, generating sales of approximately $793 million, followed closely by SRTs with combined sales of $524 million in the 7MM. This will mostly be driven by long-standing efficacy and safety profiles of ERTs and SRTs, as well as established treatment familiarity.”

*  7MM – The US, France, Germany, Italy, Spain, the UK, and Israel.

*  5EU – France, Germany, Italy, Spain, and the UK.