Homocystinuria Therapeutics Market: Introduction
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.
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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, cystinesupplemented 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
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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.