The dynamics of the Usher syndrome market are anticipated to change rapidly in the coming years owing to the anticipated launch of emerging therapies that have shown positive outcomes in clinical trials. Key players, such as LABORATOIRES THÉA, NACUITY PHARMACEUTICALS, AAVANTGARDE BIO, and others that are under phases III, II, and I of clinical development have the potential to create a significant positive shift in the Usher Syndrome market size.
LAS VEGAS, April 11, 2024 /PRNewswire/ -- DelveInsight's Usher Syndrome Market Insights report includes a comprehensive understanding of current treatment practices, Usher syndrome emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
Key Takeaways from the Usher Syndrome Market Report
- According to DelveInsight's analysis, the market size for Usher syndrome reached USD 76.48 million in 2023 across the 7MM.
- Among the 7MM, the US accounted for the highest prevalent cases of Usher syndrome in 2023, with around 21K cases; these cases are expected to increase during the forecast period.
- Leading Usher syndrome companies such as LABORATOIRES THÉA, NACUITY PHARMACEUTICALS, AAVANTGARDE BIO, ODYLIA THERAPEUTICS, ELI LILLY AND COMPANY, ATSENA THERAPEUTICS, CLEARSIDE BIOMEDICAL, and others are developing novel Usher syndrome drugs that can be available in the Usher syndrome market in the coming years.
- The promising Usher syndrome therapies in the pipeline include ULTEVURSEN, NPI-001, DUAL ADENO-ASSOCIATED VIRUS-BASED USHER SYNDROME TYPE 1B GENE THERAPY, AAV- USH1C, AK-CLRN1, and ATSN-301, among others.
- In January 2020, the FDA granted Rare Pediatric Disease (RPD) designation for QR-421a for the treatment of patients with mutations in exon 13 of the USH2A gene.
Discover which therapies are expected to grab the major Usher syndrome market share @ Usher Syndrome Market Report
Usher Syndrome Overview
Usher syndrome stands as the most prevalent deaf-blind syndrome, affecting half of those with deaf-blindness below the age of 65. This condition, rooted in genetics, manifests as a combination of hearing loss, retinopathy, and vestibular areflexia, each with varying forms and onset times. It is an autosomal recessive disorder, meaning it passes from parents to offspring, impacting the crucial senses of vision, hearing, and balance. Some cases also exhibit balance disorders and bilateral vestibular areflexia. USH is categorized into three primary clinical types - 1, 2, and 3 - each stemming from mutations in different genes and further subdivided into distinct subtypes.
The diagnosis of Usher syndrome involves detailed inquiries into the individual's medical history alongside assessments of hearing, balance, and vision. Audiologic examinations gauge the range and volume of sounds perceivable by the individual. An electroretinogram measures the retina's light-sensitive cell response to electrical stimuli. Additionally, a retinal examination is conducted to inspect the retina and other eye structures. Various tests are employed to assess vestibular (balance) function, evaluating different aspects of the balance system. Most genes linked to Usher Syndrome can be clinically tested through genetic testing.
Usher Syndrome Epidemiology Segmentation
The Usher syndrome epidemiology section provides insights into the historical and current Usher syndrome patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The Usher syndrome market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
- Total Prevalent Cases of Usher Syndrome
- Type-specific Prevalence of Usher Syndrome
- Age-specific Prevalence of Usher Syndrome
- Diagnosed and Treated Cases of Usher Syndrome
Usher Syndrome Treatment Market
Presently, there is no known cure for Usher syndrome. The most effective approach involves early detection. Handling and addressing the condition entails managing issues related to hearing, vision, and balance. Typically, strategies for addressing hearing problems encompass the use of hearing aids, assistive listening devices, cochlear implants, or alternative methods of communication. Some eye specialists suggest that a substantial dosage of vitamin A palmitate might slow down, though not stop, the advancement of retinitis pigmentosa. Other avenues such as gene therapy, medication-based treatments, and artificial organ replacements are currently at the forefront. Ongoing studies into the role of Usher proteins in the function of auditory and visual cells will play a crucial role in pinpointing the most effective target for gene replacement therapy.
Results from a lengthy clinical trial, backed by the National Eye Institute and the Foundation Fighting Blindness, suggest that Vitamin A could potentially decelerate the advancement of retinitis pigmentosa. The research indicates that adults with a prevalent type of retinitis pigmentosa might find advantages in taking a daily 15,000 IU (international units) supplement of vitamin A in its palmitate form. At present, there are limited and experimental treatments for retinal degeneration, all focused on postponing the full loss of photoreceptors.
Gene therapy appears to be a promising treatment option for retinal degeneration in Usher syndrome due to its typically monogenic recessive inheritance. Moreover, since Usher syndrome patients often have impaired hearing from birth, they can be identified early on through newborn screening programs before retinal degeneration sets in. Exploring nanoparticles as a delivery mechanism holds potential as another valuable area for research.
To know more about Usher syndrome treatment guidelines, visit @ Usher Syndrome Management
Usher Syndrome Pipeline Therapies and Key Companies
- ULTEVURSEN: LABORATOIRES THÉA
- NPI-001: NACUITY PHARMACEUTICALS
- RESEARCH PROGRAM: DUAL ADENO-ASSOCIATED VIRUS BASED USHER SYNDROME TYPE 1B GENE THERAPY: AAVANTGARDE BIO
- AAV- USH1C: ODYLIA THERAPEUTICS
- AK-CLRN1: ELI LILLY AND COMPANY
- ATSN-301: ATSENA THERAPEUTICS
- RESEARCH PROGRAM: GENE THERAPY: CLEARSIDE BIOMEDICAL
Learn more about the FDA-approved drugs for Usher syndrome @ Drugs for Usher Syndrome Treatment
Usher Syndrome Market Dynamics
The dynamics of the Usher syndrome market are expected to change in the coming years. As a frequently used modality for cancer treatment, Usher syndrome presents a huge market to any new entrant, with a huge population suffering from the disorder, easily diagnosed due to its clear clinical presentation. Advances in radiation delivery technology, such as intensity-modulated radiation therapy, can help minimize radiation-induced skin toxicity, particularly in certain tumor types like breast cancer.
Prophylactic application of a topical steroid is favored for high-risk patients, while topical steroids are commonly used for acute cases. While there is interest in topical antioxidants or oral enzymes for prevention, no clear benefit has been shown. There is a significant interest in understanding mechanismsof dermal injury to mitigate the dermatologic effects of radiation therapy, although prospective data for intervention guidance are currently limited.
Furthermore, many potential therapies are being investigated for the treatment of Usher syndrome, and it is safe to predict that the treatment space will significantly impact the Usher Syndrome market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Usher Syndrome market in the 7MM.
However several factors may impede the growth of the Usher Syndrome market. Impairment of the skin barrier function carries the risks of wound formation, loss of immune function, and infection. Further, radiation can also damage the microvascular system, increasing the risk of tissue hypoxia and fibrosis and activating an inflammatory cascade, leading to acute and chronic skin changes. Depending on the severity and location of chronic skin changes after RT, there is potential for decreased quality of life due to pain, recurrent wounds, decreased range of motion, and cosmetic dissatisfaction.
Despite technological advances in radiation delivery and growing interest in managing skin reactions, there is no gold standard in Usher syndrome management. There are limited prospective randomized data on the use of pharmacologic or supportive care agents, topical or non-topical, to prevent and treat Usher syndrome. Several off-label treatment options are currently available in the market, which will capture a huge market share and thus present tough competition to any new entrant. There has been a steady rise in alternatives for Usher syndrome in the oncology space, potentially decreasing the prevalence of Usher syndrome.
Moreover, Usher Syndrome treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Usher Syndrome market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Usher Syndrome market growth.
Usher Syndrome Market Report Metrics | Details |
Study Period | 2020-2034 |
Coverage | 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. |
Usher Syndrome Market CAGR | 10.55 % |
Usher Syndrome Market Size in 2023 | USD 76.48 million |
Key Usher Syndrome Companies | LABORATOIRES THÉA, NACUITY PHARMACEUTICALS, AAVANTGARDE BIO, ODYLIA THERAPEUTICS, ELI LILLY AND COMPANY, ATSENA THERAPEUTICS, CLEARSIDE BIOMEDICAL, and others |
Key Pipeline Usher Syndrome Therapies | ULTEVURSEN, NPI-001, RESEARCH PROGRAM: DUAL ADENO-ASSOCIATED VIRUS-BASED USHER SYNDROME TYPE 1B GENE THERAPY, AAV- USH1C, AK-CLRN1, ATSN-301, RESEARCH PROGRAM: GENE THERAPY, and others |
Scope of the Usher Syndrome Market Report
- Therapeutic Assessment: Usher Syndrome current marketed and emerging therapies
- Usher Syndrome Market Dynamics: Key Market Forecast Assumptions of Emerging Usher Syndrome Drugs and Market Outlook
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Unmet Needs, KOL's views, Analyst's views, Usher Syndrome Market Access and Reimbursement
Discover more about Usher Syndrome drugs in development @ Usher Syndrome Clinical Trials
Table of Contents
1. | Usher Syndrome Key Insights |
2. | Usher Syndrome Report Introduction |
3. | Usher Syndrome Overview at a Glance |
4. | Usher Syndrome Executive Summary |
5 | Usher Syndrome Key Events |
6 | Epidemiology and Market Forecast Methodology |
6. | Disease Background and Overview |
7. | Usher Syndrome Treatment and Management |
8. | Usher Syndrome Guidelines |
9. | Usher Syndrome Epidemiology and Patient Population |
10. | Patient Journey |
11. | Key Endpoints in Usher Syndrome |
12. | Usher Syndrome Marketed Drugs |
13. | Usher Syndrome Emerging Drugs |
14. | 7MM Usher Syndrome Market Analysis |
15. | Market Access and Reimbursement |
16. | KOL Views |
17. | Unmet Needs |
18. | SWOT Analysis |
19. | Appendix |
20. | DelveInsight Capabilities |
21. | Disclaimer |
22. | About DelveInsight |
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