Cystinosis Treatment Market 2020 Key Findings, Regional Analysis, Size, Share, Industry Growth, Key Players Profiles and Future Prospects
SEATTLE, March 29, 2021, (PHARMIWEB) — Global Cystinosis Treatment Market
Cystinosis is a condition characterized by the accumulation of cystine (amino acid) within the cells of the body. High amount of cystine damages cells, and often forms crystals that are capable of impairing several organs and tissues. The eyes and kidneys are highly susceptible to this damage; however, the pancreas, muscles, testes, and thyroid glands can also get affected. Cystinosis is categorized into three different types – nephropathic, non-nephropathic (or ocular,) and intermediate cystinosis. 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.
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All the three types of cystinosis are said to occur due to mutations in the CTNS gene. Mutations in CTNS gene cause deficiency of a transporter protein, called cystinosin. Cystinosin normally moves the cystine out of the lysosomes, which are compartments in the cell that recycle and digest materials. Defects in cystinosin, allows accumulation of cystine and forms crystals in the lysosomes and its buildup damages cells in the eyes and kidneys.
According to the National Institutes of Health (NIH), cystinosis affects at least 1 of every 100,000 to 200,000 infants around the world. Cystinosis is the most common cause of renal Fanconi syndrome in children and accounts for nearly 5% of all childhood cases of kidney failure. Fanconi syndrome, a rare disorder of kidney tubule function results in excess amounts of potassium, uric acid, glucose, phosphates, bicarbonate, and certain amino acids excreted in the urine. Cysteine depleting therapy can significantly reduce cysteine levels inside the cells. It improves growth and decreases kidney damage in children. Symptomatic therapy treats renal Fanconi syndrome with a high intake of electrolytes and fluids to prevent extreme body dehydration. Sodium bicarbonate, potassium, sodium citrate, and magnesium can help to keep standard electrolyte stability. Acetyl cholinesterase (ACE) inhibitors are also used sometimes to limit the growth of the renal disease.
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Global Cystinosis Treatment Market Insights
Various government grants to support the diagnosis and treatment of cystinosis is boosting the growth of the global cystinosis treatment 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 the Cystinosis Research Network, are responsible for propagating awareness about the disease by celebrating ‘Cystinosis Awareness Day’ on May 7, each year. Furthermore, product approvals by the Food and Drug Administration (FDA) is expected to boost growth of the global 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. In 2013, FDA approved Cystaran (cysteamine ophthalmic solution) for the treatment of corneal cystine crystal accumulation. Treatment with Cystaran should be initiated as soon as cystine crystals are found within the corneas. Lack of focus on R&D due to low prevalence rate and limited treatment options can negatively impact the global market development.
Global Cystinosis treatment Market – Regional Insights
North America is expected to spearhead the global cystinosis treatment market in the years to come. As per an article published by the 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 the disease across the region. High prevalence of cystinosis across Europe is expected to drive the growth of the cystinosis treatment market in the region. According to the National Institutes of Health (NIH), cystinosis affects around 1 per 26,000 persons in France, every single year.
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Global Cystinosis Treatment Market – Competitive Background
The key players operating in the global cystinosis treatment market include Avrobio, Inc., Gilead, Mylan N.V., Novartis AG, and Orizon Pharma Plc. These companies are currently focusing on new product development in a bid to gain a major revenue share. For instance, Avrobio Inc. initiated the clinical development program for two gene therapy candidates for cystinosis and Gaucher disease around mid-2019.
Global Cystinosis Treatment Market Taxonomy
On the basis of treatment type:
- Cysteine depleting therapy
- Symptomatic therapy
- Renal transplantation
By end user:
- Specialty Clinics
- North America
- Latin America
- Asia Pacific
- Middle East
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Table of Content
Global Cystinosis Treatment Market Research Report
Section 1: Global Cystinosis Treatment Industry Overview
Section 2: Global Economic Impact on Cystinosis Treatment Industry
Section 3: Global Market Competition by Industry Producers
Section 4: Global Productions, Revenue (Value), according to Regions
Section 5: Global Supplies (Production), Consumption, Export, Import, geographically
Section 6: Global Productions, Revenue (Value), Price Trend, Product Type
Section 7: Global Market Analysis, on the basis of Application
Section 8: Cystinosis Treatment Market Pricing Analysis
Section 9: Market Chain, Sourcing Strategy and Downstream Buyers
Section 10: Strategies and key policies by Distributors/Suppliers/Traders
Section 11: Key Marketing Strategy Analysis, by Market Vendors
Section 12: Market Effect Factors Analysis
Section 13: Global Cystinosis Treatment Market Forecast
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