Homocystinuria Therapeutics Market Size, Trends, Scope, Competitive Landscape and Forecast by 2030

Homocystinuria is a disorder of sulfur metabolism pathway caused by deficiency of cystathionine β-synthase (CBS). Increased homocysteine results in various vascular and neurological complications. Thromboembolism, affecting both large and small arteries and veins, is the major cause of morbidity and mortality. Intellectual deficiency rarely manifests before the first to second year of life. Clinically significant psychiatric illness is found in 51% of cases.

High demand for disease specific novel treatment can act as a major driver of the global market. Population studies suggest that the global prevalence of homocystinuria in the general population is 1 in 344,000. Prevalence may be higher in different countries. Europe has reported higher cases than North America and Asia Pacific. In Ireland, the prevalence has been estimated to be 1 in 65,000. In Germany, the prevalence has been estimated at 1 in 17,500.
However, limited patient pool for clinical trials and product marketing is likely to hamper the growth of the global market in the near future. Clinical trials for homocystinuria therapeutics are often smaller than those of other drugs and require the development of efficient trial designs relevant to small populations to gain the most information from the available data. Further progress is needed in trial design aiming to assess medicines in a cost-effective, rigorously controlled manner. These factors are likely to hamper the growth of the global market.

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In terms of drug treatment, the global homocystinuria therapeutics market can be classified into pyridoxine, betaine, and others
Pyridoxine is the first-line therapeutic prescribed for homocystinuria treatment. In pyridoxine non-responsive individuals, the recommended treatment is a methionine-restricted, cystine-supplemented diet in combination with the pyridoxine, folic acid, and vitamin B12 supplementation. In May 2018, Duchesnay USA launched Bonjesta (doxylamine succinate and pyridoxine hydrochloride), multilayer extended-release tablets

Based on distribution channel, the global homocystinuria therapeutics market can be divided into hospital pharmacies, retail pharmacies, and online pharmacies
The retail pharmacies segment accounted for the largest share of the global homocystinuria therapeutics market in 2019. However, the online pharmacies segment is expected to expand at the highest CAGR from 2020 to 2030.
Retail pharmacies, presently, use cloud-based inventory management to share information across several locations, automatically re-order stock, and review past transactions. In Asia, the majority of drug retailing in carried out via hospital and retail pharmacy channels. On average, retail pharmacies account for 71% of sales of therapeutic drugs, while hospital pharmacies account for 29% across the region.

In terms of region, the global homocystinuria therapeutics market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
In terms of revenue, Europe dominated the homocystinuria therapeutics market in 2019, followed by North America. However, the market in Asia Pacific is projected to expand at the highest CAGR during the forecast period.
Rise in cases of homocystinuria, surge in usage, and increase in awareness about therapeutics available are the key factors boosting the growth of the homocystinuria therapeutics market in Europe. The disorder is more common in Ireland, Germany, and Norway. Several studies in four countries in Europe suggested that the prevalence of homocystinuria due to CBS deficiency is as high as 1 in 6,400 to 1 in 20,000.

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