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Long QT Syndrome (LQTS) Market Assessment: Epidemiology, Treatment Landscape, Unmet Needs, Emerging Therapies, and Value & Access

  • Report

  • 25 Pages
  • October 2023
  • Region: Global
  • REACH Market Research
  • ID: 5996436

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
2. EPIDEMIOLOGY & PATIENT POPULATIONS
  • 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
3. DIAGNOSIS & CURRENT TREATMENT
  • 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
4. UNMET NEED
  • Overview
  • Figure 4.1. Top unmet needs in LQTS
  • High unmet need patient types
5. PIPELINE ANALYSIS
  • 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
6. VALUE & ACCESS
  • 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
7. METHODOLOGY
  • Primary market research approach
  • Epidemiology methodology
  • Long QT syndrome disease definition
  • Long QT syndrome incidence estimates
  • Long QT syndrome prevalence estimates
  • References

Samples

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Companies Mentioned

  • Thryv Therapeutics
  • Vertex