Myelodysplastic Syndrome Market Insight

DelveInsight’s ‘Myelodysplastic Syndrome - Market Insights, Epidemiology and Market Forecast– 2030’ report delivers an in-depth understanding of the Myelodysplastic Syndrome, historical and forecasted epidemiology as well as the Myelodysplastic Syndrome market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), And Japan.

The Myelodysplastic Syndrome market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Myelodysplastic Syndrome market size from 2018 to 2030. The report also covers current Myelodysplastic Syndrome treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

The United States
EU5 (Germany, France, Italy, Spain, and the United Kingdom)
Japan

Study Period: 2018–2030

Myelodysplastic Syndrome Disease Understanding

Myelodysplastic Syndrome Overview

Myelodysplastic syndrome (MDS) is a heterogeneous group of hematologic neoplasms classically described as a clonal disorder of hematopoietic stem cells leading to dysplasia and ineffective hematopoiesis in the bone marrow. In MDS, also known as myelodysplasia, the bone marrow cells do not develop into mature blood cells; instead, these cells stay within the bone marrow in an immature state. There are many subtypes of MDS; some cases are mild, while others are more severe and carry a high risk of becoming acute myelogenous leukemia (AML). The subtype and the severity case depend on many factors, including how low the blood counts are and any genetic changes in bone marrow cells. MDS and aplastic anemia (AA) and Paroxysmal nocturnal hemoglobinuria (PNH), are bone marrow failure diseases. Bone marrow failure occurs when the marrow does not produce enough red cells, white cells, or platelets, or the produced blood cells are damaged or defective. This means the body cannot supply itself with the blood it needs.

MDS may be de novo or related to prior use of chemotherapeutic agents, also known as treatment-related MDS (t-MDS). The actual preceding factor(s) for de novo MDS is not entirely understood but assumed to occur from an oncogenic process resulting in one or more somatic mutations. Over recent years, much insight has been gained into mutations that are commonly altered in MDS due to advances and the rapid availability of gene sequencing. With these developments, researchers can identify one or more driver mutations in up to 80─90% of patients with some of the most common ones, including SF3B1, TET2, SRSF2, ASXL1, DNMT3A, RUNX1, U2AF1, TP53, and EZH2. RUNX1, for example, is a mutation noted to disrupt normal hematopoiesis. More than 100 genes are recurrently mutated in MDS, and these encode spliceosome components, chromatin remodeling factors, epigenetic pattern modulators, and transcription factors, among others.

Myelodysplastic Syndrome Diagnosis

MDS is generally diagnosed when a patient is evaluated for low blood counts, although in some MDS patients, the white blood count, platelet count, or both may be elevated. The main feature of MDS is a bone marrow aspirate and biopsy that reveals heavy infiltration with abnormal-looking bone marrow cells. A chromosome analysis, called cytogenetics, is performed on the bone marrow sample. In patients with MDS, immature cells called blasts make up less than 20% of the cells in the bone marrow. If blast cells make up more than 20%, the patient is diagnosed with acute myeloid leukemia.

Myelodysplastic Syndrome Treatment

The MDS is a very heterogeneous group of myeloid disorders where the prognosis of patients with MDS can be calculated using a number of scoring systems. In general, all these scoring systems include analysis of peripheral cytopenias, percentage of blasts in the bone marrow, and cytogenetic characteristics. The most commonly accepted system is the Revised International Prognostic Scoring System (IPSS-R). Somatic mutations can help define prognosis and therapy. Therapy is generally selected based on risk, transfusion needs, and percent of bone marrow blasts, cytogenetic and mutational profiles, comorbidities, the potential for allogeneic stem cell transplantation (alloSCT), and prior exposure to hypomethylating agents (HMA). The goals of therapy are different in lower-risk patients than in higher risk and in those with HMA failure. In lower risk, the goal is to decrease transfusion needs and transformation to higher risk disease or AML and improve survival. In higher risk, the goal is to prolong survival. Currently available therapies include growth factor support, lenalidomide, HMAs, intensive chemotherapy, and alloSCT. Approved in 2020 are luspatercept and the oral HMA ASTX727. At the present time, there are no approved interventions for patients with progressive or refractory disease, particularly after HMA based therapy. Options include participation in a clinical trial or cytarabine-based therapy or alloSCT.

Myelodysplastic Syndrome Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Incident Population of MDS, Age-specific Incident Population of MDS, Subtype-specific Incident Population of MDS, Risk-specific Incident Population of MDS, and Mutation-specific Incident Population of MDS in the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan.

Key Findings

This section provides glimpse of the MDS epidemiology in the 7MM.

In 2020, the incident population of MDS in the 7MM was 41,586.
Epidemiology assessed for MDS showed that the US, in 2020, accounted for approximately 20,428 incident cases of MDS.
In the United States, in 2020, there were 240, 1,070, 3,654, 7,283 and 8,181 cases, in the age groups 0–49 years, 50–59 years, 60–69 years, 70–79 years, and 80+ years, respectively.
As per the estimates, in the United States, there were 6,741, 1,021, 2,043, 6,333, 3,064, and 1,226 cases of RAEB/MDS-EB, del(5q) MDS, MDS-RS/RARS, MDS-MLD/RCMD, MDS-SLD/RCUD, and MDS-U subtypes in 2020, respectively.
In 2020, there were 4,903, 2,043, 817, 2,043, 2,554, 1,839, 4,086, 2,860, 817, and 1,021 cases, for the mutation SF3B1, SRSF2, U2AF1, DNMT3A, RUNX1 TP53, TET2, ASXL1, ZRSR2, and IDH2 respectively, in the US.
In the US, based on the IPSS scoring, Low-risk MDS and High-risk MDS accounted for the highest and lowest cases, respectively, in 2020. On the other hand, based on the IPSS-R scoring, Low-risk MDS and Very high-risk MDS accounted for the highest and lowest cases, respectively, in 2020.
Among the EU-5 countries in 2020, Germany had the highest incident population of MDS patients with 4,515 cases, followed by France (4,371) and UK (3,302). In contrast, Spain had the lowest cases (2,010) in 2020.
As per the estimates, Japan accounted for 3,909 incident MDS cases in 2020.

Country Wise- Myelodysplastic Syndrome Epidemiology

The epidemiology segment also provides the Myelodysplastic Syndrome epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Myelodysplastic Syndrome Drug Chapters

The drug chapter segment of the Myelodysplastic Syndrome report encloses the detailed analysis of MDS mid and late-stage pipeline drugs. It also helps to understand the MDS clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.

Myelodysplastic Syndrome Marketed Drugs

Reblozyl: Bristol-Meyers Squibb

Reblozyl (luspatercept-aamt) is an erythroid maturation agent indicated for the treatment of anemia failing an erythropoiesis-stimulating agent and requiring two or more RBC units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). Bristol Myers Squibb and Acceleron Pharma are jointly developing Reblozyl as part of a global collaboration.