Arrhythmogenic Cardiomyopathy (U.S.), October 2023
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: Gain insights into the drug pricing landscape and payer controls within the disease market or analogous markets.
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.
1. DISEASE OVERVIEW
A life-threatening, progressive inherited genetic condition impacting cardiac function
Table 1.1. Most common ACM mutations
Figure 1.1. Pathogenesis of ACM
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Prevalence of ACM
Table 2.1. ACM diagnosed prevalence in the United States, 2023
Figure 2.1. Prevalence estimates of ACM in the medical literature from 1994 – 2022
Disease definition
Figure 2.2. Diagnosed prevalent cases of PKP2+ ACM by region
Table 2.2. Diagnosed prevalent ACM cases in the US and EU5
Table 2.3 Average prevalence of ACM
mutation type
3. DIAGNOSIS AND CURRENT TREATMENT
Diagnosis overview
Figure 3.1. Diagnostic pathway for ARVC patients
Figure 3.2. Physician-reported average estimate of ACM patients that receive a diagnosis
ACM genetic testing dynamics
Figure 3.3. KOL estimate of their ACM patients who are PKP2+ with genetic testing
Figure 3.4. Physician-reported barriers to genetic testing
ACM treatment involves pharmacological and device interventions
Figure 3.5. Treatment algorithm for management of ACM
Figure 3.6. ACM treatment goals
ACM treatment dynamics
Figure 3.7. Current treatment patient share
Table 3.1. Current ACM treatment outcomes
Table 3.2. Upsides and downsides of current interventions used for ACM
Key treatment dynamics that shape disease management and novel therapy use in ACM
Table 3.3. Must-know ACM market dynamics
Key treatment dynamics that shape disease management and drug use in ACM
Figure 3.8. Important dynamics of ACM market evolution1
4. UNMET NEED
Overview
Figure 4.1. Top unmet needs in ACM
Figure 4.2. Physician-reported perspectives on ACM treatment
ACM disease burden and unmet needs
Figure 4.3. Physician-reported disease burden for ACM patients and families
Table 4.1. Most important attributes for new ACM treatment
Physician perspectives on unmet needs in ACM
5. PIPELINE ANALYSIS
Overview
Table 5.1 Summary of the RP-A601 gene therapy trial
ACM therapies in early pipeline follow similar framework
Table 5.2. Comparison of preclinical ACM gene therapies
Investigator-sponsored drug-based approaches to ACM
Figure 5.1. Estimated proportion of population with positive AAV titers
Table 5.3. Investigator-sponsored ACM trials
Gene therapy in ACM
Table 5.4. Physician-reported perspectives on ACM treatment and gene therapies
Table 5.5. Attributes that will drive uptake for emerging ACM therapies
Future considerations for gene therapy
Table 5.6. Opportunities and threats for emerging AAV-based ACM gene therapies
Physician perspectives on ACM gene therapies
6. VALUE & ACCESS
Overview
Table 6.1. Comparison of gene therapy pricing, U.S.
Figure 6.1. Key reimbursement and access considerations for emerging therapies in ACM
Key insights from the ACM market
Table 6.2. Innovative payer coverage gene therapy insights
7. METHODOLOGY
Primary market research approach
Epidemiology methodology
Table 7.1. ACM prevalence and population size references
PKP2 mutation rate methodology
Table 7.2. Mutation rate references
Table 7.3. Pharmaceutical company ACM and PKP2 prevalence estimates
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