MarketVue® Report
Angelman Syndrome (U.S.), 2024

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Angelman Syndrome (U.S.), February 2024

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The MarketVue®: Angelman Syndrome 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®: Angelman Syndrome report is supported by 8 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:
  • Roche
  • Ionis
  • Biogen
  • Ultragenyx
  • Neuren Pharmaceuticals
  • Foundation for Angelman Syndrome Therapeutics (FAST)
  • Encoded Therapeutics
  • Transformatx
  • Ovid Therapeutics
 
Key drugs mentioned:
  • Melatonin
  • Trazodone
  • Clonazepam
  • Lamotrigine
  • Levetiracetam
  • Topiramate
  • Clonidine
  • Atomoxetine (Strattera)
  • Risperidone
  • Aripiprazole (Abilify)
  • Polyethylene glycol
  • Omeprazole
  • Rugonersen
  • ION582
  • GTX-102
  • NNZ-2591
  • Alogabat
  • GTP-220
  • Gaboxadol
1. DISEASE OVERVIEW
A rare, neurogenetic disorder characterized by developmental delays, seizures, and a happy and excitable demeanor
Table 1.1. AS molecular subtypes
The role of UBE3A in the disease mechanism of AS
Figure 1.1. The role of UBE3A in the hypothesized pathogenesis of AS
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Disease definition
Figure 2.1. G6 diagnosed prevalent cases of Angelman Syndrome by region
Table 2.1. Diagnosed incident and prevalent populations of AS in the U.S. and EU5
AS epidemiology and subtypes
Figure 2.2. Proportion of AS patients with each molecular subtype
3. DIAGNOSIS & CURRENT TREATMENT
Diagnosis overview
Figure 3.1. Diagnostic pathway for AS patients
AS requires lifelong medication courses and therapy
Table 3.1. Treatment goals for AS – physician versus caregiver priorities
Figure 3.2. Proportion of AS patients with difficult-to-treat seizures
AS treatment involves a multidisciplinary care team collaborating with caregivers
Figure 3.3. AS care team roles and responsibilities
AS key characteristics throughout life course
Physician insights on current AS treatment approaches
Key treatment dynamics that will shape disease management and novel therapy use in AS
Table 3.2. Must-know AS market dynamics
Large opportunity for a therapy that will address neurodevelopmental outcomes
Figure 3.4. Important dynamics of AS market evolution
4. UNMET NEED
Overview
Table 4.1. Top unmet needs in AS
Physician perspectives on unmet needs in AS
5. PIPELINE ANALYSIS
Overview
Table 5.1. Comparison of ongoing AS disease-modifying therapy clinical trials
Pipeline overview continued
Table 5.2. Comparison of ongoing AS non-disease-modifying therapy clinical trials
Preclinical AS gene therapy and delivery platforms
Figure 5.1. Novel “HItap” AAV delivery platform
Table 5.3. Preclinical GTP-220 summary
Preclinical AS gene therapy pipeline
Table 5.4. Preclinical research on AS gene therapies
Physician insights on gene therapy options
Failed AS trials and important drivers of future AS product differentiation
Figure 5.2. Ovid Therapeutics OV101 Phase 3 efficacy data
Important drivers of future AS product differentiation
Figure 5.3. Attributes that will drive uptake for emerging AS therapies
6. VALUE & ACCESS
Overview
Table 6.1. Comparison of gene therapy pricing
Overview continued
Table 6.2. Comparison of ASO therapy pricings
Figure 6.1. Key reimbursement and access considerations for emerging gene therapies in AS
Key insights from the gene therapy market
Table 6.3. Innovative payer coverage gene therapy insights
Financial hurdles in AS diagnosis and treatment
Figure 6.2. Mean annual financial impact on caregivers for AS
Figure 6.3. Mean annual healthcare costs per patient with development epileptic encephalopathies
7. METHODOLOGY
Primary market research approach
Epidemiology methodology
Table 7.1. Diagnosed prevalent populations of AS by age
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