IDH1-mutated Relapsed or Refractory Myelodysplastic Syndromes (MDS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034

 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 – 2024 To 2034" 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 (MDS) 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 coverage:

G8 (United States, EU5 [France, Germany, Italy, Spain, U.K.], Japan, and China)

Insights driven by robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support

*As per Thelansis’s policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2024–2034)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across G8 markets (US, EU5, Japan, and China)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market’s trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?
• and more…

Read more: IDH1-mutated Relapsed or Refractory Myelodysplastic Syndromes (MDS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034