Cystinosis Treatment Market Boosting The Growth: Market Dynamics And Trends, Efficiencies Forecast 2026 With Avrobio, Inc., Gilead, Mylan N.V., Novartis AG, and Orizon Pharma Plc

Cystinosis is a condition characterized by accumulation of the cystine (amino acid) within cells. High amount of cystine damages cells and often forms crystals that can damage tissues and organs. The eyes and kidneys are mainly susceptible to damage; however, the pancreas, muscles, testes, and thyroid can also be affected. Cystinosis is categorized into three different types; nephropathic, non-nephropathic or ocular, and intermediate cystinosis.

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Nephropathic (infantile) cystinosis is the most severe and common form of cystinosis. Renal Fanconi syndrome and growth failure are usually the first noticeable symptoms of this disorder. Intermediate cystinosis is characterized by symptoms similar to that of nephropathic cystinosis. However, these symptoms are not prominent till age of 8-20 years. Non-nephropathic cystinosis generally affects adults during middle age with eye disorder. Kidney disease does not occur in these individuals. Individuals with non-nephropathic cystinosis develop photophobia due to cystine crystal accumulation in the eyes.

Cystinosis Treatment Market Dynamics

Various government grants to support the diagnosis and treatment of cystinosis is boosting growth of the market. For instance, in 2013, the Cystinosis Research Foundation awarded six grants worth US $1.29 million to Cystinosis researchers for developing better treatments and a cure for the disease. Organizations such as Cystinosis Research Network are responsible to spread awareness regarding disease by celebrating Cystinosis Awareness Day on May 7, every year.

Furthermore, product approval by the Food and Drug Administration (FDA) is expected to boost growth of the cystinosis treatment market over the forecast period. In 2013, the FDA approved the delayed-release formulation of cysteamine bitartrate (DR-C) for patients aged 6 years and above, and the label was expanded in 2015 to include children age two years. Cysteamine is a cystine-depleting agents that reduces cystine levels in cells. It has proven to be effective in preventing or delaying renal failure and improving growth of children with Cystinosis.

Cystinosis Treatment Market Competitive Landscape

Key players operating in the cystinosis treatment market include Avrobio, Inc., Gilead, Mylan N.V., Novartis AG, and Orizon Pharma Plc. Companies in the cystinosis treatment market are focusing on new product development to gain major revenue share. For instance, Avrobio Inc. is planing to initiate clinical development of two gene therapy candidates for cystinosis and Gaucher disease by mid-2019.

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Cystinosis treatment Market Regional Insights

Regional segmentation of Cystinosis treatment market by Coherent Market Insights, includes North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. North America is the expected to dominate the cystinosis treatment market. According to an article published by Cystinosis Research Network, an estimated 15 new nephropathic cystinosis cases are diagnosed in the U.S. annually. Moreover, the network also works on increasing awareness about disease among regional population. High prevalence rate of cystinosis in Europe is expected to drive growth of the cystinosis treatment market in the region. According to National Institutes of Health (NIH), cystinosis affects 1 in 26,000 persons in France.

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