IDH1-mutated Relapsed or Refractory Myelodysplastic Syndromes (MDS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

IDH1-mutated Relapsed or Refractory Myelodysplastic Syndromes (MDS) Market Outlook

Thelansis’s “IDH1-mutated Relapsed or Refractory Myelodysplastic Syndromes (MDS) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential IDH1-mutated Relapsed or Refractory Myelodysplastic Syndromes treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

IDH1-mutated Relapsed or Refractory Myelodysplastic Syndromes (MDS) Overview

The gene Isocitrate dehydrogenase 1 (IDH1) encodes the protein isocitrate dehydrogenase 1, an enzymatic component of the citric acid cycle. This enzyme facilitates the carboxylation process of isocitrate, leading to alpha-ketoglutarate formation. Recurrent mutations in the IDH1 gene have been observed in 12% of glioblastoma patients and 70% of patients diagnosed with WHO grade II and III astrocytomas and oligodendrogliomas. Within these contexts, the IDH1 mutations target a specific amino acid residue at position 132, causing a substitution from arginine to histidine (R132H). The clinical significance of IDH1 mutations in patients with myelodysplastic syndromes (MDS) remains inadequately understood. The IDH1 enzyme is synthesized based on the genetic instructions present in the IDH1 gene, located on chromosome 2q33.3. The enzyme is situated in both the cytoplasm and peroxisomes. Ordinarily, the IDH1 gene encodes an enzyme reliant on NADPH that facilitates the transformation of isocitrate into alpha-ketoglutarate. In the presence of IDH1 mutations, the enzyme's functionality is altered, producing the oncometabolite D2HG and subsequent accumulation. In acute myeloid leukemia (AML), the most frequently identified IDH1 mutation occurs at the Arg132 residue (R132). This alteration results in substituting the substrate-binding arginine with residues such as R132H, R132C, R132G, R132L, or R132S in the catalytic domain of the enzyme. The outcomes of R132 IDH1 mutations include DNA and histone hypermethylation and a hindrance in cellular differentiation, indicating the presence of leukemogenic myeloid progenitor cells. Both IDH1 and IDH2 mutations are correlated with older age and an unfavorable prognosis, particularly in AML cases characterized by a cytogenetically normal karyotype (CN-AML). Various other factors are associated with IDH1 mutations, including intermediate-risk cytogenetics, elevated platelet counts, higher percentages of bone marrow blasts during diagnosis, mutations in FLT3-ITD and NPM1, and, in rare instances, mutations linked to therapy-related AML, TET2, and WT1. Multiple methodologies, such as Sanger sequencing, PCR-based techniques, and next-generation sequencing (NGS), are employed to identify IDH mutations in patients with hematologic malignancies. Patients with IDH1-mutated relapsed or refractory MDS currently have no targeted therapy options, and outcomes are generally poor for those who experience disease progression after treatment with standard care.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2022-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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