Alport Syndrome (U.S.), June 2022
The MarketVue®: Alport Syndrome market landscape report combines primary (KOL interviews and survey data) and secondary market research to empower strategic decision-making and provide a complete view of the market.
Every MarketVue® includes a disease overview, epidemiology (US and EU5), current treatment, unmet needs, pipeline and access and reimbursement chapter.
Topics covered in this report:
- Disease overview: Review the disease pathophysiology and potential druggable targets
- Epidemiology: Understand prevalence, diagnosed and drug-treated prevalence of the population and key market segments
- Current treatment: Understand the treatment decision tree and strengths and weaknesses of current on-label and off-label treatment
- Unmet needs: Identify opportunities to address treatment or disease management gaps
- Pipeline analysis: Compare current and emerging therapy clinical development strategy; their performance on efficacy, safety, and delivery metrics; and their potential to address unmet needs
- Value and access: Gain insights into the drug pricing landscape and payer controls within the disease market or analogous markets
Methodology:
Research for the MarketVue®: Alport Syndrome report is supported by 3 qualitative interviews with key opinion leaders (U.S. nephrologists), a quantitative survey with 25 U.S. physicians and secondary research.
Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom)
Key companies mentioned:
- Reata Pharmaceuticals
- Sanofi
- Chinook Therapeutics
- River 3 Renal Corp
- Travere Therapeutics
Key drugs mentioned:
- Angiotensin-receptor blockers (ARBs)
- Spironolactone
- Eplerenone (Inspra)
- Dapagliflozin (Farxiga)
- Empagliflozin (Jardiance)
- Bardoxolone
- Lademirsen
- Atrasentan
- Lisinopril (Prinivil, Zestril)
- Sparsentan (Filspari)
- R3R01
- Ramipril (Altace)
1. DISEASE OVERVIEW
A progressive, inherited nephropathy
Alport syndrome progresses in four phases
Figure 1.1. Phases of Alport syndrome disease progression
There are three types of Alport syndrome
Table 1.1. Types of Alport syndrome
Figure 1.2. Percent of XLAS patients that progress to ESRD by age and sex
Figure 1.3. Modes of inheritance for Alport syndrome
Hearing loss and eye abnormalities in Alport syndrome
Figure 1.4. Percentage of nephrologists who “often-to-always” coordinate patient care with other specialists
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Disease definition
Figure 2.1. G7 diagnosed prevalent cases of Alport syndrome by region
Table 2.1. Diagnosed prevalent and drug-treated patients in the U.S. and EU5
Treatment rates are high although AS is underdiagnosed
Figure 2.2. Percentage of key patient subpopulations – mode of inheritance and gender
Patients have a significant influence over the evolving treatment landscape of AS
3. CURRENT TREATMENT
Drug-treated patients and treatment rates
Table 3.1. Treatment initiation and rates by risk of disease progression
Figure 3.1. Percentage of nephrologists initiating treatment with ACEis/ARBs in Alport patients
Current standard of care is to achieve RAS blockade and blood pressure control with ACEis and ARBs
Standard of care – Blood pressure-lowering medicines
Figure 3.2. Current treatment share by patient subtype
Figure 3.3. Nephrologists’ opinion on deciding treatment between ACEis and ARBs
Nephrologists’ opinion on current treatments for Alport syndrome
Current treatment algorithm for Alport syndrome
Figure 3.4. Diagnosis and treatment flow for Alport syndrome
Nephrologists’ opinion on currently available treatments
Figure 3.5. Standard of care – upside and downside
Figure 3.6. Nephrologist-reported percentage of patients with unsatisfactory outcomes on current treatments
Almost all nephrologists see AS patients
Treating physicians and their patient load
Coordination of care for Alport syndrome
Figure 3.7. Percentage of nephrologists coordinating with other specialists
Disease awareness continues to be low, affecting timely treatment
Current and future must-know treatment dynamics
Figure 3.8. Percentage of nephrologists citing low disease awareness as a significant barrier to Alport syndrome diagnosis among GPs and other nephrologists
AS market likely to remain unchanged in the next few years
Figure 3.9. Potential Timeline of Alport Syndrome Market Evolution
4. UNMET NEED
Treatment-related unmet needs
Figure 4.1. Top unmet needs in Alport syndrome
Figure 4.2. Nephrologist ranking of unmet needs in Alport syndrome
Patients want a disease-targeting therapy that prevents progression to renal failure
Alport syndrome patients’ insights on unmet needs
Figure 4.3. Percentage of patients experiencing various negative effects of Alport Syndrome on daily living
Low disease awareness among GPs is a significant diagnostic barrier for AS
Barriers to diagnosis
Figure 4.4. Percentage of nephrologists rating barrier as significant in timely diagnosis of Alport syndrome patients
5. PIPELINE ANALYSIS
Overview
Figure 5.1. Percentage of nephrologists with aided awareness of emerging therapies for Alport syndrome
Table 5.1. Emerging clinical-stage therapies for Alport syndrome and nephrologist awareness
Nephrologists see endothelin receptor antagonists as promising targets
Figure 5.2 Percentage of U.S. nephrologists rating target as “promising”
Basket trials are becoming a more common way to study drugs targeting Alport syndrome
Comparison of ongoing trials in Alport syndrome
Table 5.2 Comparison of clinical stage Phase 2 or 3 studies in Alport syndrome
Bardoxolone Case Study: Overview and issues raised during FDA review
Regulatory review of bardoxolone
Figure 5.3. Key issues identified during FDA review of bardoxolone
Bardoxolone Case Study (Cont): KOL perspective on clinical issues identified during FDA review
Bardoxolone Case Study (Cont): Broader implications and patient reactions
6. VALUE & ACCESS
Overview
Value & access analogue: an innovative approach in acute hepatic porphyria
Figure 6.1. Prevalence-based adjustments
Value & access analogue: a traditional approach in sickle cell disease
7. METHODOLOGY
Primary market research methodology
Epidemiology methodology
Disease definition
Diagnosed prevalence estimates
Drug-treatment rates
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