Global Cystinosis Market Size, Share & Trends Analysis Report By End-user, By Region, and Segment Forecasts, 2023 - 2032

Global Cystinosis Market Size, Share & Trends Analysis Report By End-user, By Region, and Segment Forecasts, 2023 - 2032

Global Cystinosis Market Size, Share & Trends Analysis Report By End-user, By Region, and Segment Forecasts, 2023 – 2032

Global Cystinosis Market was valued at US $169 Million in 2022 and is expected to reach ~US $300.1 Million by 2032, growing at a CAGR of 6.59% during the forecast period 2023-2032.

It is anticipated that the market for cystinosis would expand rapidly in the upcoming years due to factors like rising awareness of the condition, better rates of diagnosis, and the creation of innovative treatment options. An overabundance of the amino acid cystine within cells causes a condition known as cystinosis, which can damage cells and cause crystals to develop that can injure tissues and organs. Though the pancreas, muscles, testes, and thyroid can also be affected, the eyes and kidneys are the main organs at risk. There are three different forms of cystinosis: ocular (non-nephropathic), nephropathic (infantile), and intermediate. The most severe and common type of cystinosis is nephropathic cystinosis, which usually manifests as growth failure and renal Fanconi syndrome at first. While the symptoms of intermediate cystinosis are similar, they could not show up until a person is between the ages of 8 and 20. Middle-aged persons are affected by non-nephropathic cystinosis, which mostly causes ocular diseases without kidney involvement. Cysteine crystal buildup in the eyes causes photophobia, or sensitivity to light.

Mutations in the CTNS gene cause deficiencies in the transporter protein called cystinosin, which is the cause of all three forms of cystinosis. Normally, cystinosin makes it easier for cystine to be taken out of lysosomes, which are parts of cells that recycle and degrade things. Defective cystinosin causes cystine to build up and crystallise inside lysosomes, which eventually damages kidney and ocular cells. The National Institutes of Health (NIH) estimates that 1 in 100,000 to 200,000 infants globally are affected by cystinosis. It is responsible for around 5% of all paediatric kidney failure cases and is the primary cause of renal Fanconi syndrome in children. The uncommon illness known as Fanconi syndrome impairs kidney tubule function and causes the urine to excrete potassium, uric acid, glucose, phosphates, bicarbonate, and certain amino acids excessively. Cysteine depletion therapy can minimise renal impairment in children by increasing growth and dramatically lowering cystine levels within cells. Symptomatic therapy uses sodium bicarbonate, potassium, sodium citrate, and magnesium along with electrolytes and fluids to keep the body from severely dehydrating and to maintain the right electrolyte balance. In certain cases, acetylcholinesterase (ACE) inhibitors may also be used to slow the advancement of renal disease.

“Cystine-Depleting Therapy segment, by treatment type, to be largest market from 2023 to 2030.”

The segmentation analysis of the treatment type segment is as Cystine-Depleting Therapy and Symptomatic Therapy. Finding characteristic symptoms (such those linked to renal Fanconi syndrome), a thorough clinical examination, a patient's medical history, and a number of specialised tests are all necessary for making a cystinosis diagnosis.

Because targeted cysteamine therapy is available, patients' clinical results are greatly impacted by early diagnosis and management of cystinosis. For cystinosis, there are three main methods of diagnosis. The gold standard for illness diagnosis at the moment is looking for increased cystine levels in white blood cells (WBCs), which has remarkable sensitivity and accuracy. Molecular testing of the relatively compact CTNS gene, which has 12 exons but only 10 of them are transcribed, is another well-established method. 95% of mutations that cause disease may be found using this method. Slit-lamp examination is the third confirming method used in clinical practice to identify distinctive corneal cystine crystals.

“Hospitals segment, by end user, to be largest market from 2023 to 2030.”

The global Cystinosis market by end user comprises major segments such as hospitals, specialty clinics and others. Hospitals are essential to the care and treatment of cystinosis. They offer complete medical care, which includes inpatient treatment, diagnostics, emergency services, and specialised care for patients with severe cystinosis-related symptoms or sequelae. Multidisciplinary teams in hospitals are frequently equipped to handle many facets of this illness, including kidney failure and visual indications. Complex or uncommon diseases like cystinosis are managed exclusively in speciality clinics. They provide more specialised and targeted therapy, frequently offering patients long-term treatment regimens, follow-up, and support. Experts in nephrology, ophthalmology, and other pertinent fields may be found in speciality clinics, providing cystinosis patients with more specialised and all-encompassing care. This category may include different service providers or providers not included in the hospital or speciality clinic categories. Home health care services, research facilities carrying out cystinosis-related clinical trials or studies, and nonprofit groups offering assistance, resources, and advocacy to patients and their families are a few examples.

“North America to be largest region in Cystinosis Market.”

There are chances for manufacturers in North America to grow their businesses as a result of the growing demand for cystinosis therapy in the healthcare industry. There are many manufacturers and suppliers in North America, and the country's quick economic development has increased industrial production, which in turn has increased demand for cystinosis treatment.

The rise in the cystinosis treatment market share in North America can be attributed to various factors, including rising healthcare costs, an increase in research endeavours, technological developments in the treatment of metabolic disorders, and the opening of pharmaceutical and biotechnology companies in the area.

The need for cystinosis therapy in the area is predicted to increase as a result of cooperative research projects between significant healthcare organisations and commercial enterprises. The market is expanding in part because of continuous efforts to improve current treatment choices through new product development. The market in this region is expected to grow further as people become more aware of other treatment options, such as cysteine-depleting therapy for management purposes. Taken together, these elements highlight North America's leading position in the world market for cystinosis treatments. With the United States leading the way, North America is still a major force in the worldwide cystinosis treatment market. The region's increased need for cystinosis therapy is a result of government policies supporting investment, enhancing infrastructure, and emphasising healthy lifestyles. The demand for cystinosis treatment has been further increased by the aggressive implementation of numerous initiatives and research activities, particularly by the United States.

Cystinosis Competitive Landscape

The competitive landscape of the Cystinosis Market involves assessing the competitive landscape to understand the strengths, weaknesses, opportunities, and threats of the industry. Key industry players have recognized that the adoption of Cystinosis technology holds the potential for further growth. The growing desire among producers to optimize their production costs has spurred collaborative efforts among companies to scale up their production capacity. This strategic collaboration not only aims to increase revenue but also seeks to establish dominance in the market.

The Cystinosis Market is highly competitive, with numerous companies vying for market share. Prominent companies in the Cystinosis Market include:

Major Players:

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

Biophore.

Recent Developments:

As of June 12, 2023, AVROBIO, Inc.'s investigational gene therapy for cystinosis, based on the haematopoietic stem cell (HSC) system, has been sold to Novartis, as per the previously disclosed agreement. The top clinical-stage gene therapy provider verified the conclusion of this deal, in which Novartis purchased the treatment for a cash payment of $87.5 million. Remarkably, AVROBIO continues to be the exclusive owner of its ground-breaking HSC gene therapies for Gaucher disease types 1 and 3, Pompe illness, and Hunter syndrome.

The UZH research team began using the PandaOmics platform on July 14, 2023, in an effort to find previously approved drugs that might be used as cystinosis treatments. This thorough investigation included a review of the target enzymes, the structural characteristics of the medications, any possible side effects, and how well they worked in the impacted tissues. In the process, rapamycin—a drug that is already approved—became a very promising candidate for treating cystinosis. Rapamycin therapy has been shown to be able to preserve cellular processes and restore lysosomal function in model species and cell systems.