Long QT Syndrome (LQTS)

MarketVue®: Long QT Syndrome (LQTS)

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 by REACH, 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.""

Please note: the online download version of this report is for a global site license.