The MarketVue®: Glycogen Storage Disease Type 1a 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®: GSD1a report is supported by 7 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:
- Ultragenyx
- Moderna
- Beam Therapeutics
Key drugs mentioned:
- DTX-401
- mRNA-3745
- BEAM-301 / huR83C
Key takeaways from the report:
GSD-1a, also known as von Gierke's disease, is a rare inherited disorder that hinders the body's ability to break down glycogen, a stored form of sugar, leading to a daily risk of hypoglycemia, which can result in seizures and in extreme cases, death. There are currently no FDA approved therapies for GSD-1a.
In the absence of approved therapies, the condition requires patients to consume raw cornstarch every 3-4 hours to ensure a sustained glucose supply, coupled with the constant need for glucose monitoring. This regimen leads to chronic sleep disruption and anxiety for both patients and caregivers. With a lifestyle described by physicians as 'not conducive to normal living', patients must adhere to severely restricted diets and have no choice but to consume the majority of their caloric intake from precisely measured amounts of cornstarch.
Endocrinologist, U.S.: 'The issue is it's hard to be perfect, and people get tired of the treatment, which is every three to four hours around the clock, then people stop being compliant, and then they get into trouble.'
An AAV8 vector gene therapy, DTX-401, is currently in phase 3 clinical trials and will likely be the first drug treatment for the disease if approved by the FDA. However, DTX-401's durability is currently unknown, and may not be the one-time solution patients believe gene therapy to be given the inability to redose the treatment. Pre-existing AAV antibodies also render some patients ineligible for this type of gene therapy, and the prevalence of antibodies among the GSD-1a patient population is unknown.
Endocrinologist, U.S.: 'I don't think doctors realize that gene therapy doesn't treat everything. Doctors don't realize that it will run out at some point, and then we have to figure out what we're going to do after that.'
The GSD-1a pipeline beyond DTX401 is limited to two other gene therapies in development. These include Moderna's intravenous mRNA therapy, mRNA-3745, currently in phase 1/2, and Beam Therapeutics' BEAM-301, a preclinical DNA base editor. These emerging therapies hold potential for expanding the treatment options available to individuals with GSD-1a.
Table of Contents
1. DISEASE OVERVIEW- A rare, autosomal recessive disease preventing metabolism of glucose and resulting in glycogen accumulation in the body’s cells
- Figure 1.1. GSD-1a metabolic impairment in the cytoplasm of the liver and kidney cells
- GSD-1a symptoms and complications
- Genetic variants of GSD-1a
- Table 1.1. GSD-1a mutation variations
- Disease definition
- Table 2.1 Diagnosed prevalent and incident populations of GSD-1a in the U.S. and EU5
- Table 2.2. Prevalent population considerations
- Diagnosis overview
- Figure 3.1. Diagnostic pathway for GSD-1a patients
- Genetic testing access and considerations for GSD-1a diagnosis
- Treatment goals
- Figure 3.2. Treatment goals for GSD-1a1
- Figure 3.3. Pillars of treatment for GSD-1a
- GSD-1a dietary treatment algorithm
- Figure 3.4. Dietary treatment algorithm for the management of GSD-1a
- Upsides and downsides of cornstarch
- Figure 3.5. Upsides and downsides of cornstarch
- GSD-1a treatment algorithm continued
- Figure 3.6. Treatment algorithm for the management of GSD-1a
- Figure 3.7. Physician-reported average % of adult GSD-1a patients with adenomas
- Physician insights on current GSD-1a treatment approaches
- Key treatment dynamics that will shape disease management and novel therapy use in GSD-1a
- Table 3.1. Must-know GSD-1a market dynamics
- Large opportunity for a therapy that will reduce burdens of managing GSD-1a
- Figure 3.8. Important dynamics of GSD-1a market evolution
- Overview
- Figure 4.1. Top unmet needs in GSD-1a
- Physician perspectives on unmet needs
- Overview
- Table 5.1. Comparison of ongoing clinical trials for emerging GSD-1a gene therapies
- Pipeline overview continued
- Figure 5.1. Figure 5.1 Ultragenyx AAV8 DTX-401 efficacy data
- Table 5.2. Pre-clinical GSD-1a gene therapies
- Efficacy, eligibility, and durability will be important drivers of future GSD-1a product differentiation
- Figure 5.2. Attributes that will drive uptake for emerging GSD-1a therapies
- Further treatment eligibility considerations
- Table 5.3. Attributes that will drive uptake for emerging GSD-1a therapies
- Figure 5.3. Proportion of people w/ positive AAV titers
- Physician insights on gene therapy mechanisms
- Overview
- Table 6.1. Comparison of gene therapy pricing, U.S.
- Figure 6.1. Key reimbursement and access considerations for emerging gene therapies in GSD-1a
- Key insights from the GSD-1a market
- Table 6.2. Innovative payer coverage gene therapy insights
- Insurance hurdles in GSD-1a diagnosis and treatment
- Figure 6.2. Mean annual healthcare costs per patient by healthcare resource
- Primary market research approach
- Epidemiology methodology
- Epidemiology methodology cont.
Samples
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Companies Mentioned
- Ultragenyx
- Moderna
- Beam Therapeutics
