The Gaucher disease market is expected to grow at a compound annual growth rate (CAGR) of 0.79% from 2023 to 2032, reaching $1.3bn across the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Israel) by the end of this forecast period, according to GlobalData’s recent report, Gaucher Disease: Opportunity Assessment and Forecast. The major factors driving growth include the increase in Gaucher disease patients in line with population growth, improved diagnostic protocols, and the launch of venglustat malate which is the first brain-penetrant substrate reduction therapy (SRT).

Gaucher disease is a rare inherited metabolic disorder characterised by a deficiency of the enzyme glucocerebrosidase (GCase) leading to harmful lipid accumulation. Common manifestations include hepatosplenomegaly (abnormally enlarged liver and spleen), thrombocytopenia (low platelet levels), anaemia (low red blood cell levels), and skeletal abnormalities. These manifestations of Gaucher disease vary across its types: type 1 (non-neuronopathic), type 2 (acute neuronopathic), and type 3 (chronic neuronopathic).

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Current therapies involve enzyme replacement therapy (ERT) to boost GCase levels, while SRT aims to lower glucocerebroside levels. While ERTs and SRTs are effective for treating systemic symptoms commonly displayed by type 1 patients, interviews with key opinion leaders (KOLs) highlighted the serious lack of effective therapies for treating neuropathic manifestations, typically affecting type 2 and type 3 patients. While the Gaucher disease market is anticipated to grow during the forecast period, KOLs emphasised the need for more convenient and less invasive therapies, as well as improved physician awareness and diagnostic methods. Early diagnosis could enable physicians to prevent the development of irreversible symptoms such as advanced bone disease to improve patient outcomes and reduce strain on healthcare systems.

Looking ahead, the Gaucher disease drug pipeline offers a combination of innovative products and therapies which emulate marketed treatments. Currently in Phase III, Sanofi’s venglustat malate is an SRT that GlobalData expects to capitalise on the market success of Actelion’s Zavesca and Sanofi’s Cerdelga, both approved SRTs for type 1 patients. Sanofi’s innovative therapy option is believed to penetrate the central nervous system and potentially relieve neuropathic symptoms such as poor coordination and seizures. Expected to launch in the US and five major European markets (5EU: France, Germany, Italy, Spain, and the UK) within the 2023–32 forecast period, GlobalData reports that venglustat malate may leverage the trend of younger patients transitioning from ERT to SRT, positioning itself to cater to this evolving demographic seeking convenient therapy options. As such, GlobalData projects that venglustat malate will secure a substantial share of the Gaucher disease market upon its launch.

Although the late-stage pipeline is relatively restricted, the early-stage gene therapies in development, most notably Freeline Therapeutics’ FLT-201 and Prevail Therapeutics’ PR-001, have demonstrated promise in Phase II. Poised to offer the greatest potential to impact the Gaucher disease landscape, these gene therapies may provide curative, long-lasting therapy options for patients by addressing the underlying genetic mutations and eliminating the need for a lifetime of injections and oral tablets. While these gene therapies take advantage of adenovirus-associated vector mechanisms, which have proven successful in treating spinal muscular atrophy and haemophilia, there is still significant work ahead to establish their efficacy and safety profiles for Gaucher patients.

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With steady market growth anticipated throughout the forecast period, pharmaceutical companies are urged to persist in the development of affordable, minimally invasive brain-penetrant therapies and to foster meaningful dialogues with patient organisations, ensuring sustained progress and improved outcomes for patients with Gaucher disease.

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