MarketVue®: Arrhythmogenic Cardiomyopathy
The MarketVue®: Arrhythmogenic Cardiomyopathy 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®: Arrhythmogenic Cardiomyopathy report is supported by 13 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:• Rocket Pharmaceuticals
• Lexeo Therapeutics
• Tenaya Therapeutics
• StrideBio / Ginko Bioworks
• Ginkgo Bioworks
• BioMarin Pharmaceuticals
Key drugs mentioned:• Beta blockers
• Sotalol (Betapace, Sorine)
• ACE inhibitors
• RP-A601
• TN-401
• STRX-330
• LX2020
• BMN365
• Spironolactone
• Flecainide (Tambocor)
Key takeaways from the report:
Arrhythmogenic Cardiomyopathy (ACM) is a rare hereditary cardiac disorder characterized by progressive replacement of myocardial tissue with fibrofatty deposits, leading to life-threatening arrhythmias and cardiac dysfunction.
ACM patients typically receive treatment with beta blockers and anti-arrhythmic drugs to manage symptoms and arrhythmias. However, according to REACH Market Research’s MarketVue® assessment, a striking 50% of patients face treatment failure, necessitating invasive procedures like implantable cardioverter-defibrillators (ICDs), catheter ablation, or even heart transplants which coincide with their own set of challenges.
The urgency for innovative treatments targeting the underlying disease pathophysiology to slow down or halt the progression of the disease, ultimately reducing the risk of life-threatening cardiac events is clear, however, the disease exhibits incomplete penetrance, making risk prediction and patient stratification challenging.
Cardiologist, U.S.: ""[There is] the inability to actually prevent the underlying progression since there are no medications that will prevent progression or reduce the cause of arrhythmias, so even if they've got an ICD, they may get shocked from it or have poor quality of life.""
The ACM pipeline remains sparse, with two gene therapies entering early-stage clinical development targeting the PKP2 gene, the most prevalent ACM mutation:
• Rocket Pharmaceuticals’ AAVrh74-based gene therapy is in Phase 1
• Lexeo Therapeutics is on track to initiate a Phase 1/2 trial for their AAVrh10-based gene therapy following IND clearance in August 2023
These developments offer new hope to ACM patients, however, given the disease can be caused by genetic mutations outside of PKP2, these treatments would target less than half of diagnosed prevalent patients.
Please note: the online download version of this report is for a global site license.