The MarketVue®: Long QT Syndrome (LQTS) market landscape report combines primary (KOL interviews) 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: Review the evidence needed to assess and communicate value to key stakeholders (e.g., providers, payers, regulators) and learn what competitors have done or are doing
Methodology:
Research for the MarketVue®: Long QT Syndrome report is supported by 10 qualitative interviews with key opinion leaders and secondary research.
Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom).
Key companies mentioned:
- Thryv Therapeutics
- Vertex
Key drugs mentioned:
- LQT-1213
- Lumacaftor / Ivacaftor (Orkambi)
Key takeaways from the report:
Long QT syndrome (LQTS) is a rare congenital cardiac arrhythmia characterized by a prolonged recovery in cardiac action potential that can cause deadly arrhythmias.
According to LQTS experts interviewed, most cases of LQTS, especially LQTS type 1, are well-controlled by the current standard of care - beta blockers - despite less the less-than-ideal side effects they cause (e.g., fatigue, depression, decreased libido). However, for high-risk patients that experience breakthrough events despite maximal tolerated beta blocker treatment, physicians turn to surgical options that include implantable cardioverter-defibrillators (ICDs) or left cardiac sympathetic denervation (LCSD). While surgical options are largely effective at controlling symptoms (LCSD) or reliably correcting potentially fatal arrhythmias (ICD), these approaches are used as a last resort and come with notable limitations such as:
- ICDs, especially when implanted in children, have a high risk of malfunction and require continued maintenance surgeries like battery replacement
- LCSD recovery can be very painful and there is a shortage of surgeons trained to perform LCSD
Consequently, there is a need for additional pharmaceutical treatments to reduce the reliance upon surgical options for refractory patients. Additionally, while a large majority of patients are well-managed by beta blockers, up to half of these patients struggle with the side effects and could benefit from a better-tolerated treatment option. Unfortunately for LQTS patients, the clinical pipeline is sparse, with only one industry-sponsored drug in development therefore it will likely be 5 or more years before any new treatments become available.
Cardiac Electrophysiologist, U.S.: 'I have a bunch of ICD patients who have abandoned leads or who have had multiple lead extraction procedures that are high risk, there's been a perforation, and they had to have a sternotomy and have gone really, really bad. A lot of those are my patients with long QT syndrome who have had an ICD since childhood.
Table of Contents
1. DISEASE OVERVIEW- LQTS subtypes
- Table 1.1. Comparison of the three most common LQTS subtypes
- Disease definition
- Figure 2.1. Diagnosed prevalent cases of LQTS by region
- Incidence of LQTS
- Prevalence of LQTS
- Diagnosis and drug treatment rates
- Table 2.1 Diagnosed incident and prevalent LQTS cases in the US and EU5
- Most common subtypes of LQTS
- Figure 2.2. Proportion of LQTS patients with the three most common subtypes
- Table 2.2. Description of LQTS subtypes
- Diagnosis overview
- Figure 3.1. Diagnosis flow of LQTS patients
- Risk stratification is an important prognostic tool for LQTS
- Figure 3.2. Factors considered in patient risk status
- LQTS has evolved from an often-deadly diagnosis to a chronically managed condition
- Treatment overview
- Figure 3.3. Treatment goals for LQTS - physician versus patient priorities
- Treatment flow for LQTS
- Figure 3.4. Treatment algorithm for LQTS
- Comparison of approved treatments for LQTS
- Figure 3.5. Comparison of approved treatments for LQTS
- Opportunity for better treatments exist for certain groups of high-risk or intolerant patients
- Figure 3.6. KOL estimates of the proportion LQTS patients that are poorly managed
- Table 3.1. Standard of care - upside and downside
- Disease burden and key treatment dynamics that shape disease management in LQTS
- Table 3.2. Must-know LQTS treatment dynamics for now and the future
- No significant changes are anticipated to disrupt the LQTS market in the foreseeable future
- Figure 3.7. Important dynamics of LQTS market evolution
- Overview
- Figure 4.1. Top unmet needs in LQTS
- High unmet need patient types
- Looking backward
- Looking forward
- Figure 5.1. Evolution of pharmaceutical targets for LQTS
- There is only one industry sponsored clinical trial in LQTS
- Table 5.1.Comparison of active trials for LQTS
- Physician insights on various potential LQTS therapies
- Opportunity for novel therapy in LQTS
- Overview
- Table 6.1. Comparison of treatment pricing, U.S.
- Pricing analogue; camzyos (mavacamten) for hypertrophic cardiomyopathy
- Table 6.2. Typical U.S. commercial payer coverage
- Primary market research approach
- Epidemiology methodology
- Long QT syndrome disease definition
- Long QT syndrome incidence estimates
- Long QT syndrome prevalence estimates
- References
Companies Mentioned
- Thryv Therapeutics
- Vertex
