Summary

Overview
Global cystinosis treatment market reached US$ xx billion in 2022 and is expected to reach US$ xx billion by 2030, growing with a CAGR of xx% during the forecast period 2023-2030.
The global cystinosis treatment market has witnessed significant growth and transformations over the years, with various factors influencing its dynamics. Population growth, research and development and government investments in pharmaceuticals are among the key drivers impacting cystinosis treatment globally. After the challenging year of 2020, the cystinosis treatment industry showed moderate resilience and adaptability.
Government investments in metabolic disorders, particularly in developing economies, will continue to drive cystinosis depleting therapy and boost the global cystinosis treatment market. The global cystinosis treatment industry is placing increasing emphasis on better management of cystinosis. The respective initiatives includes the use of alternative treatments, such as growth hormone therapy, vitamin D supplements or fluid replacement therapy to reduce or treat the symptoms of cystinosis.
The nephropathic cystinosis type of cystinosis, accounts for over half of the market share. Similarly, the North America dominates the cystinosis treatment market, capturing the largest market share of over 1/3rd. The region’s rapid investments in healthcare and rising major players in the cystinosis treatment market globally, makes it the largest market share holder. United States has the largest pool of manufacturers as well as consumers.
Dynamics
Growing Research and Development by the Researchers
Developed economies have been witnessing rapid growth in betterment of their healthcare sectors, driven by rising income levels, urbanization and infrastructure development. Several countries have experienced substantial demand for cystinosis treatment, reflecting their expanding healthcare industries. Rising research and development will be a crucial factor driving the growth of the market.
In Jul 2023, the UZH researchers investigated the molecular process behind kidney illness in cystinosis in collaboration with Insilico Medicine, a business that employs AI for medication discovery. They discovered the infectious pathways and selected targets for therapy within cystinosis cells by utilizing model systems with Insilico's PandaOmics platform. Their research showed a connection between the disease and the control of a protein termed mTORC1.
According to the study, cystine accumulation causes the mTORC1 protein to become activated, impairing kidney tubular differentiation and function of cells. There is a pressing need for more efficient therapies since individuals with cystinosis frequently need kidney transplants to restore renal function. There is a pressing need for more efficient therapies since individuals with cystinosis frequently need kidney transplants to restore renal function. The UZH study group therefore started looking for current medications that may be used to treat cystinosis using the PandaOmics platform.
Rising Number of Clinical Trials with Positive Outcomes
One of the primary drivers of the global cystinosis treatment market is robust pipeline, especially in emerging economies and rising clinical trials with positive outcomes. For instance, on May 18, 2023, follow-up data from a collaborator-sponsored Stage 1/2 clinical trial assessing an investigational gene therapy approach for the management of cystinosis show a durable therapeutic impact across important indicators out to 36 months, according to AVROBIO, Inc., an established clinical-stage gene treatment company dedicated to rescuing people from an entire life of genetic disease.
These findings were presented in Los Angeles, California, on May 18, 2023, at the 26th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. Measurements in several tissues, such as the skin, gastrointestinal mucosa, eyes, and brain, and also by neurocognitive tests that are suggestive of CNS activity. To date, there have been no reports of significant adverse events (SAEs) or adverse events (AEs) connected to the drug product.
Adverse Effects Associated with the Cystamine Depletion Therapy
Rare adverse reactions to cysteamine at higher dosages have included bone pain, skin striae, myalgia, and elbow lesions that resemble bruises (reactive angioendotheliomatosis upon skin biopsy). Cysteamine improves the survival of human dermal microvascular endothelial cells in a lab setting, which could help to explain why it causes angioendotheliomatosis. Treatment is also connected to an offensive sulfurous body and breath odor when it is converted into volatile sulfuric compounds (i.e., methanethiol and dimethyl sulfide), which frequently results in low treatment compliance.
Other adverse effects of cysteamine include hyperthermia, allergic rash, lethargy, seizures, neutropenia, and gastrointestinal unease; however, these side effects are reversible, and the majority are avoided by starting the medication at a low dosage and gradually increasing it as tolerated with the help of skilled medical professionals.
Less Knowledge Among People Leading to Complications owing to the Delay in Treatment
As less people have knowledge about cystinosis treatment hence leading to delay in treatment which can create lot of complications. Kidney failure will set in for cystinosis patients by the age of ten without therapy. Many kids experience renal failure in late childhood or adolescence, often despite receiving treatment.
Dialysis and eventually a kidney transplant will be necessary because of this. Infantile nephropathic cystinosis individuals that go untreated develop corneal cystine crystal buildup that causes ocular pain and photophobia. Cysteine eye drops are employed to dissolve crystals and alleviate symptoms because cysteamine treatment taken orally has no impact on corneal cystine crystals.
A novel topical viscous mixture for cysteamine drops for the eye has been created that is more effective than traditional cysteamine eye drops while requiring less frequent instillation. Numerous neurologic issues, such as anatomical abnormalities of the brain, cognitive decline, and learning challenges are related to cystinosis. Long-term research has suggested that starting cysteamine oral therapy early may help to prevent several of these neurocognitive problems.
Segment Analysis
The global cystinosis treatment market is segmented based on type, treatment, distribution channel and region.
High Incidence Rate of Nephropathic Cystinosis
The cystine transporter cystinosin, a widely expressed lysosomal cystine-proton co-transporter, is found at the lysosomal membrane and regulates the cystine efflux from the lysosome. Cystinosis is thought to affect 1 in 100,000 to 200,000 live births. The most prevalent and severe clinical manifestation of the disease, infantile nephropathic cystinosis, accounts for 95% of all cases and is marked by substantial morbidity and death.
Cystinosis is an extremely uncommon lysosomal storage disorder brought on by autosomal recessive alterations in the CTNS gene. Mutations in the CTNS gene result in a lack of cystinosin or its absence, which causes lysosomes to accumulate free cystine and form poisonous crystals, both of which can harm tissue and organs.
As a result of the buildup of corneal cystine crystals, cystinosis is a metabolic condition that first affects the kidneys. It then spreads to the endocrine and reproductive systems, skin, muscles, lungs, bones, and the central nervous system. Although it is not considered a curative approach, a fundamental component of medication is a depletion therapy using cysteamine that has been shown to slow down the advancement of renal failure and to avoid or slow down extra-renal symptoms.
Geographical Penetration
North America Accounted for Largest Market Share in 2022, Owing to the Strong Presence of Major Players and Increasing Healthcare Infrastructure Investment
Due to the rising need for cystinosis treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand for cystinosis treatment.
Increasing expenditure on healthcare and rising research studies, advancement of technologies for treatment of metabolic diseases, and increase in pharmaceutical and biotechnology business establishment across the region are also contributing to the growth of cystinosis treatment market share of this region.
The key healthcare organizations' and businesses' joint research initiatives are also expected to contribute to the rising demand, as will new product development, which constantly looks to improve present options. The market in this area is growing as people become more aware of various treatments, such as cysteine depleting therapy, for management purposes. The aforementioned elements further attest to North America's hegemonic position in the world.
North America continues to be a key player in the global cystinosis treatment market, with United States leading the way. Government initiatives promoting infrastructure development and investment, and a focus on healthy lifestyle have fueled the demand for cystinosis treatment in the region. United States have been proactive in executing several initiatives or researches, stimulating cystinosis treatment demand.
COVID-19 Impact Analysis
The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global cystinosis treatment market. As countries grappled with lockdowns, supply chain disruptions and reduced economic activity, the healthcare sector, which is a significant consumer of cystinosis treatment, was significantly impacted.
The onset of the pandemic in early 2020 led to widespread lockdowns and restrictions, impacting several activities worldwide. Major pharmaceutical or biotechnology industries came to a standstill and shifted their attention towards the management of COVID-19, leading to a slump in demand for cystinosis treatment. As a result, many pharmaceutical plants either suspended or reduced production to align with the reduced demand.
The COVID-19 pandemic severely disrupted global supply chains, impacting the transportation of raw materials to the pharmaceuticals manufacturing plants. Movement limitations and border closures caused production and supply delays. Additionally, some nations experienced a lack of raw resources as a result of supply chain network interruptions.
With the outbreak of the pandemic, many pharmaceutical manufacturing units faced labor shortages as workers fell sick or were unable to travel to work due to lockdown restrictions. To curb the spread of the virus, pharmaceutical plants implemented stringent safety measures, reducing the number of workers allowed on-site at a time. However, these measures were vital to safeguard the health of the workforce and maintain essential operations during the pandemic.
Russia-Ukraine War Impact Analysis
The pharmaceutical businesses are starting to worry regarding their capacity to continue operating, much like other parts of the world. Russia's invasion of Ukraine has an effect on millions of people not only in this nation in Eastern Europe but also all around the world. Drug developers will thus experience delays in the development process, a loss of business consistency, and the possibility of non-compliance for drugs already on the market.
For many years, clinical trials in several therapeutic fields have benefited significantly from the participation of Ukraine, Russia, and other CIS nations. Clinical trial durations can frequently be accelerated by incorporating more patients into studies when there is availability to skilled investigators, high-quality patient populations, and big patient populations. Trials that are delayed in Russia and Ukraine as well as relocation to other regions of Europe for fresh trials are currently viable choices. These factors will impact the global cystinosis treatment market.
By Type
• Nephropathic Cystinosis
• Intermediate Cystinosis
• Non-Nephropathic Cystinosis
By Treatment
• Cystine-Depleting Therapy
o Cystagon
o Procysbi
o Cystaran
o Cystadrops
• Symptomatic Therapy
o Fluid Replacement
o Oral Calcium
o Growth Hormone Therapy
o Thyroxine Supplements
• Others
By Distribution Channel
• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies
By Region
• North America
o U.S.
o Canada
o Mexico
• Europe
o Germany
o UK
o France
o Italy
o Spain
o Rest of Europe
• South America
o Brazil
o Argentina
o Rest of South America
• Asia-Pacific
o China
o India
o Japan
o Australia
o Rest of Asia-Pacific
• Middle East and Africa
Key Developments
• On June 12, 2023, the previously reported agreement to sell AVROBIO, Inc.'s investigational gene therapy based on hematopoietic stem cell (HSC) system for cystinosis to Novartis for $87.5 million in cash has been closed, reported by the leading clinical-stage gene therapy company. Full ownership of AVROBIO's first-in-class HSC gene treatments for Pompe disease, Hunter syndrome, and Gaucher disease types 1 and 3 remains with the company.
• On July 14, 2023, the UZH research team started looking for existing medications that could be used to treat cystinosis using the PandaOmics platform. Analysis of the target enzymes, drug's structure, potential adverse effects, and effectiveness in the affected tissues were all part of this process. A promising possibility for the treatment of cystinosis has been found as the already-approved medication rapamycin. Studies on model species and cell systems demonstrated that rapamycin therapy restored lysosome functioning and saved cellular processes.
Competitive Landscape
The major global players in the market include Leadiant Biosciences, Inc., Recordati Rare Diseases, Horizon Therapeutics plc, CHIESI Farmaceutici S.p.A., BioSpectra, Inc., VIVAN Life Sciences, Viatris Inc., 3S Corporation, Merck KGaA and Biophore among others.
Why Purchase the Report?
• To visualize the global cystinosis treatment market segmentation based on type, treatment, distribution channel and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of cystinosis treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.
The global cystinosis treatment market report would provide approximately 61 tables, 58 figures and 186 Pages.
Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

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