MarketVue® Report
Sorbitol Dehydrogenase Deficiency (SORD Deficiency) (U.S.), 2022

Syndicated market assessment report driven by primary market research

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Sorbitol Dehydrogenase Deficiency (SORD Deficiency) (U.S.), March 2022

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The MarketVue®: Sorbitol Dehydrogenase (SORD) Deficiency 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), 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®: Sorbitol Dehydrogenase (SORD) Deficiency report is supported by 8 qualitative interviews with key opinion leaders and secondary research.
 
Geographies covered:
United States
 
Key companies mentioned:
  • Applied Therapeutics
 
Key drugs mentioned:
  • AT-007
1. DISEASE OVERVIEW
SORD deficiency awareness
Figure 1.1. Neuromuscular specialist familiarity with SORD Deficiency
Figure 1.2. Percentage of neurologists familiar with role of sorbitol in peripheral neuropathy
Grading disease severity in CMT
Figure 1.3. Proportion of physicians using CMT severity scales to grade disease severity
Table 1.1. Description and utility of validated CMT disease severity scoring scale
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Prevalence of SORD deficiency
Figure 2.1. SORD deficiency prevalence and population size in the United States, 2022
Figure 2.2. Global prevalence estimates of CMT, 1974 – 2019
Patient flow calculation of the number of SORD deficiency patients
Figure 2.3. Derivation of SORD deficiency population size in the United States, 2022
Figure 2.4. SORD deficiency population segmented by disease severity
3. DIAGNOSIS & CURRENT TREATMENT
Diagnosis overview
Figure 3.1. Diagnosis flow CMT2 and SORD deficiency patients
Delayed diagnosis of CMT disorders
Figure 3.2. Drivers of delayed diagnosis in CMT2 patients
Genetic testing in CMT
Figure 3.3. Genetic testing use and results in CMT2 patients
Figure 3.4. Summary of Invitae’s Comprehensive Neuropathies Panel
Genetic re-testing for SORD deficiency
Figure 3.5. Percentage of neuromuscular specialists who re-test patients for newly identified genes
Figure 3.6. Neuromuscular specialists’ use of sorbitol testing now and in the future
Treatment overview
Figure 3.7. Treatment goals for CMT2 disorders
Table 3.1. Standard of care – upside and downside
Treatment dynamics in CMT2
Table 3.2. Key CMT2 treatment dynamics that will impact SORD deficiency now and in the future
4. UNMET NEED
Overview
Figure 4.1. Physician-reported unmet needs in CMT2 patients
5. PIPELINE ANALYSIS
Overview
Table 5.1 Summary of AT-007 INSPIRE study
Figure 5.1 Key considerations for emerging therapies for SORD Deficiency
6. VALUE & ACCESS
Insurance coverage of CMT2 patients
Overview of drug analogues
Figure 6.1. Average percentage of CMT patients covered by insurance type among interviewed physicians
Table 6.1. Criteria for selecting SORD deficiency analogues
Commercial payer requirement of drug analogues
Table 6.2. Summary of commercial insurance requirements for Soliris and Oxlumo
Figure 6.2. Important market access considerations for SORD deficiency drug developers
Alnylam’s market access pillars for Oxlumo
Figure 6.3. Alnylam market access strategies to promote access to Oxlumo in the United States
Drug Analogue: Soliris in PNH
Figure 6.4. Annual cost of Soliris (000s), 2007 – 2021
Figure 6.5 Key market access pillars for Soliris in PNH
7. METHODOLOGY
Primary market research approach
Table 7.1. Participant screening criteria
Figure 7.1. Interviewed neuromuscular specialists segmented by types of patients seen (number of physicians)
Diagnosed prevalence estimates approach
Disease definition
Table 7.3. Key population dynamics references
Table 7.4. CMT prevalence references
Table 7.5. dHMN prevalence references
Table 7.6. CMT subtype prevalence references
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