DelveInsight’s “Cystinosis Market Insight, Epidemiology and Market Forecast 2032” report provides current treatment practices, emerging drugs, Cystinosis market share of the individual therapies, current and forecasted Cystinosis market size from 2019 to 2032 segmented by seven major markets. The report also offers current Cystinosis therapy algorithms, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the Cystinosis market.
Key Takeaways from the Cystinosis Market Report
- According to DelveInsight estimates, the Cystinosis market size was found to be USD 174 million in 2021.
- Globally, key Cystinosis companies such as Avrobio, Mylan, Horizon Pharma, Chiesi Farmaceutici, Recordati S.p.A, Leadiant Biosciences, and others are developing novel Cystinosis drugs that can be available in the Cystinosis market in the upcoming years.
- Promising Cystinosis therapies in the pipeline include AVR-RD-04 (CTNS-RD-04), A0003, and others.
- The increase in market size is a direct consequence of the increasing prevalent population of Cystinosis patients in the 7MM.
Discover more about therapies set to grab major Cystinosis market share @ Cystinosis Market Report
Cystinosis Overview
Cystinosis is a disease caused by an abnormal buildup of cystine, an amino acid. It is a rare but serious disease with long-term consequences that can affect many body parts. Cystinosis is classified into three types based on when symptoms first appear (known as onset): infantile (early-onset) cystinosis, late childhood or adolescent (late-onset) cystinosis, and adult cystinosis.
Early-onset cystinosis is the most severe type. Cystinosis symptoms, complications, and timing can vary depending on the type of disease. Cystinosis is diagnosed using various methods, including a physical exam to look for disease symptoms. If cystine crystals are found in the eyes, they will be examined (signs of eye problems appear after age).
Learn more about the Cystinosis treatment guidelines @ Cystinosis Guidelines
Cystinosis Epidemiological Insights
In the assessment done by DelveInsight, the estimated total diagnosed prevalent cases of Cystinosis in the 7MM were approximately 1,347 in 2021.
Among 7MM, the United States has the highest diagnosed prevalent cases of cystinosis, with approximately 665 cases in 2021. In EU-5, the UK has the highest number of cases, i.e., 224, followed by Germany, with 182 cases. While Italy has the least number of cases, with 43 in 2021.
The Cystinosis market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:
- Total Prevalent Cases
- Diagnosed Prevalent Cases
- Type-specific Diagnosed Prevalent Cases
Download the report to understand which factors are driving Cystinosis epidemiology trends @ Cystinosis Epidemiological Insights
Cystinosis Treatment Market
There is currently no cure for Cystinosis, and treatment is limited to a few approved therapies that provide symptomatic relief. In the United States, three therapies are currently approved, one for ocular cystinosis and the other two for nephropathic cystinosis.
There is currently only one upcoming therapy in the Cystinosis pipeline, AVR-RD04 (Gene therapy), which will assess the long-term safety and durability of AVR-RD-04 treatment in participants who received a single dose administration of lentiviral gene therapy and will further reduce the market share of both drugs. The upcoming gene therapy will target the entire Cystinosis patient population, and it is expected to hit the market in 2026, according to our estimates. The introduction of this therapy will reduce the market for all existing drugs and is expected to generate significant revenue.
To know more about Cystinosis treatment options, visit @ Cystinosis Drugs
Cystinosis Pipeline Therapies
- AVR-RD-04 (CTNS-RD-04)
- A0003
Cystinosis Key Companies
- Avrobio
- Mylan
- Horizon Pharma
- Chiesi Farmaceutici
- Recordati S.p.A
- Leadiant Biosciences
Learn more about the Cystinosis therapies in clinical trials @ New Treatment for Cystinosis
Cystinosis Market Dynamics
The dynamics of the cystinosis market are anticipated to change in the coming years. With increasing cases and awareness, more companies are expected to focus on the therapeutic pipeline related to Cystinosis. Moreover, Procysbi was initially approved for patients aged 6 years and older. Later in 2015, it received extended approval for children aged 2 to 6, and in 2017, it received approval for children aged 1 and up. And it is currently being investigated for different dose formulations, with the FDA accepting the submitted NDA for the new dose.
Procysbi’s recommended starting dosage is divided into two doses administered every 12 hours. Cystagon, on the other hand, is administered in four divided doses. As a result, most patients have shifted their treatment away from cystagon toprocysbi. The benefit is obvious: patients maintain a normal sleep interval and do not require a mid-day dose. This demonstrates that Procysbi is targeting a large patient pool of patients of various ages. If its new dosage formulation is accepted, it will help increase its cystinosis market share.
However, numerous types of side effects are associated with the currently widely used cysteamine therapy, including halitosis, disagreeable sweat odor, and gastrointestinal side effects. Frequent dosing has a direct negative impact on the quality of life of patients and families. There are currently only two oral cysteamine therapies approved for nephropathic cystinosis. If gene therapy is approved, research shows that it can be proven to be an effective treatment option. And the one gene therapy that is expected to be launched in the forecasted period, AVR-RD-04, has the potential to compete with Procysbi.
Scope of the Cystinosis Market Report
- Study Period: 2019–2032
- Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
- Key Cystinosis Companies: Avrobio (NASDAQ: AVRO), Mylan (NASDAQ: VTRS), Horizon Pharma (NASDAQ: HZNP), Chiesi Farmaceutici, Recordati S.p.A (BIT: REC), Leadiant Biosciences, and others
- Key Cystinosis Therapies: AVR-RD-04 (CTNS-RD-04), A0003, and others
- Therapeutic Assessment: Cystinosis current marketed and emerging therapies
- Cystinosis Market Dynamics: Cystinosis market drivers and barriers
- Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
- Unmet Needs, KOL’s views, Analyst’s views, Cystinosis Market Access and Reimbursement
Discover more about Cystinosis drugs in development @ Cystinosis Clinical Trials
Table of Contents
| 1. | Cystinosis Market Key Insights |
| 2. | Cystinosis Market Report Introduction |
| 3. | Cystinosis Market Overview at a Glance |
| 4. | Cystinosis Market Executive Summary |
| 5. | Disease Background and Overview |
| 6. | Cystinosis Treatment and Management |
| 7. | Cystinosis Epidemiology and Patient Population |
| 8. | Patient Journey |
| 9. | Cystinosis Marketed Drugs |
| 10. | Cystinosis Emerging Drugs |
| 11. | Seven Major Cystinosis Market Analysis |
| 12. | Cystinosis Market Outlook |
| 13. | Potential of Current and Emerging Therapies |
| 14. | KOL Views |
| 15. | Cystinosis Market Drivers |
| 16. | Cystinosis Market Barriers |
| 17. | Unmet Needs |
| 18. | SWOT Analysis |
| 19. | Appendix |
| 20. | DelveInsight Capabilities |
| 21. | Disclaimer |
| 22. | About DelveInsight |
Get in touch with our Business executive @ Healthcare Due Diligence Services
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