#luspatercept

[creativeera]

The Evolution of MDS Treatment: A Cross-Regional Examination of Advances in the U.S., Europe, and China

Myelodysplastic Syndrome (MDS) represents a diverse group of bone marrow disorders characterized by ineffective blood cell production and an increased risk of leukemia. As research and technology advance, treatment approaches for MDS have evolved significantly, reflecting regional differences in healthcare systems, available therapies, and patient needs. This blog explores the evolution of MDS treatment across the U.S., Europe, and China, highlighting key advancements and regional approaches that are shaping the future of care for this complex condition.

The Evolution of MDS Treatment: A Global Overview

The management of MDS has seen remarkable progress over the past few decades. Initially, treatment options were limited to supportive care and basic chemotherapy. However, the landscape has transformed with the development of targeted therapies, advancements in genetic understanding, and improved supportive care strategies. Understanding how these advancements are applied across different regions provides insight into the global approach to managing MDS.

Advances in the U.S.

In the United States, the approach to treating MDS has evolved through a combination of cutting-edge research, personalized medicine, and a focus on patient-centered care.

1. Personalized Risk Stratification

One of the most significant advancements in the U.S. has been the development of risk stratification systems such as the International Prognostic Scoring System (IPSS) and its updated versions. These tools categorize patients based on factors like genetic mutations, blood cell counts, and chromosomal abnormalities, guiding treatment decisions. The move towards personalized treatment plans allows for more tailored therapies, improving patient outcomes and minimizing unnecessary side effects.

2. Hypomethylating Agents

The introduction of hypomethylating agents, such as azacitidine and decitabine, has been a game-changer in U.S., Europe, and China Myelodysplastic Syndrome (MDS) Treatment. These drugs work by modifying DNA methylation patterns, which can lead to improved blood cell production and reduced disease progression. They are now a standard treatment for patients with intermediate to high-risk MDS, offering a significant improvement over traditional chemotherapy.

3. Targeted Therapies and Clinical Trials

The U.S. is at the forefront of developing and testing new therapies through extensive clinical trials. Advances in genetic research have led to the identification of specific mutations that drive MDS, resulting in targeted therapies designed to address these genetic abnormalities. For example, drugs like lenalidomide are used for patients with deletion 5q, a specific genetic abnormality associated with MDS.

4. Stem Cell Transplantation

Allogeneic stem cell transplantation remains one of the most promising treatment options for high-risk MDS patients. The U.S. has developed sophisticated transplantation techniques and supportive care measures to improve patient outcomes and reduce complications associated with the procedure. However, due to the high risk and complexity of stem cell transplants, this option is generally reserved for younger, healthier patients.

Advances in Europe

In Europe, the treatment of MDS has evolved with a focus on standardized guidelines, integration of new research, and access to innovative therapies. The European approach emphasizes both risk-based treatment and inclusion in clinical trials.

1. European LeukemiaNet (ELN) Guidelines

The ELN provides comprehensive guidelines for the diagnosis and treatment of MDS, which are widely adopted across European countries. These guidelines incorporate risk stratification tools similar to those used in the U.S. and offer a framework for managing MDS based on the latest research and clinical evidence.

2. Access to New Therapies

European countries have been proactive in incorporating new therapies into clinical practice. For example, hypomethylating agents and targeted therapies are widely used in Europe, with many countries following similar protocols to those in the U.S. However, variations in drug availability and healthcare policies can impact the accessibility of these treatments.

3. Clinical Trials and Research

Europe is known for its robust clinical trial network, which provides patients with access to innovative treatments that may not yet be available elsewhere. European researchers are also involved in international collaborations, contributing to the global understanding of MDS and the development of new therapies.

4. Supportive Care and Palliative Options

European healthcare systems place a strong emphasis on supportive care and palliative options for MDS patients. This includes a focus on managing symptoms, improving quality of life, and providing psychological support. This approach ensures that patients receive comprehensive care, addressing both the physical and emotional aspects of living with MDS.

Advances in China

China's approach to treating MDS reflects a blend of traditional practices and modern medical advancements. The rapid development of healthcare infrastructure and increasing investment in medical research are driving significant changes in MDS treatment in the region.

1. Improved Diagnosis and Access to Care

China has made strides in improving the diagnosis of MDS through enhanced awareness and better access to diagnostic tools. Efforts to increase awareness and improve early detection are critical in a country with a large and diverse population.

2. Integration of Western Medicine and Traditional Chinese Medicine (TCM)

In China, there is a growing interest in combining Western medical treatments with Traditional Chinese Medicine (TCM). Some patients use TCM practices such as herbal remedies and acupuncture alongside conventional therapies to manage symptoms and improve overall well-being. While TCM is not considered a cure for MDS, it offers complementary benefits that can enhance the patient’s quality of life.

3. Access to Modern Therapies

China has seen improvements in access to modern treatments like hypomethylating agents and targeted therapies. The availability of these drugs can vary depending on the region, but overall, there has been progress in making these advanced treatments more accessible to patients.

4. Stem Cell Transplantation and Research

China has a large stem cell donor registry and has made significant advancements in stem cell transplantation. This procedure is increasingly available to MDS patients, particularly those with high-risk disease. Additionally, China’s investment in research is contributing to the development of new therapies and treatment strategies for MDS.

Common Challenges and Future Directions

Despite advancements in MDS treatment across the U.S., Europe, and China, there are common challenges that need to be addressed globally:

Access to Care: Disparities in access to advanced treatments and supportive care remain a challenge, particularly in low-resource settings or rural areas. Efforts to improve access and equity in healthcare are essential for ensuring that all patients receive the care they need.

Cost of Treatments: The high cost of modern therapies, including hypomethylating agents and stem cell transplantation, can be a barrier to treatment for some patients. Addressing the cost of care and exploring ways to make treatments more affordable are important considerations.

Research and Innovation: Continued investment in research is crucial for developing new therapies and improving treatment outcomes. International collaboration and sharing of research findings can accelerate progress and lead to more effective treatments for MDS.

The treatment of Myelodysplastic Syndrome (MDS) has evolved significantly, with advancements driven by research, innovation, and regional healthcare practices. The U.S., Europe, and China each contribute unique approaches to managing MDS, reflecting their distinct healthcare systems and patient needs. As global research and collaboration continue to advance, the future of MDS treatment looks promising, with new therapies and strategies on the horizon. By learning from each region’s experiences and addressing common challenges, the global medical community can work towards improving outcomes and quality of life for individuals living with MDS.

Get more insights on U.S., Europe, And China Myelodysplastic Syndrome (MDS) Treatment

Alice Mutum is a seasoned senior content editor at Coherent Market Insights, leveraging extensive expertise gained from her previous role as a content writer. With seven years in content development, Alice masterfully employs SEO best practices and cutting-edge digital marketing strategies to craft high-ranking, impactful content. As an editor, she meticulously ensures flawless grammar and punctuation, precise data accuracy, and perfect alignment with audience needs in every research report. Alice's dedication to excellence and her strategic approach to content make her an invaluable asset in the world of market insights.

(LinkedIn: www.linkedin.com/in/alice-mutum-3b247b137 )

[mezzopieno-news]

UNA NUOVA TERAPIA CURA IL TUMORE DEL SANGUE

Ogni anno 5.800 persone sono colpite in Italia da mieloma multiplo, un tumore del sangue che ha origine nel midollo osseo. Negli ultimi venti anni la sopravvivenza media è passata da 36 mesi a 7 anni.

Una nuova terapia cellulare ha evidenziato un tasso di risposta completa nel 45,9% dei pazienti recidivanti con mieloma multiplo. Nel linfoma a grandi cellule B, liso-cel, la nuova CAR T, ide-cel ha dimostrato la scomparsa di tutti i segni di malattia nel 74% dei casi refrattari. Il nuovo immunomodulante orale, Iberdomide, ha permesso di raggiungere risposte globali del 36,8% nei pazienti pesantemente pretrattati aprendo anche prospettive importanti nel linfoma non-Hodgkin, con un miglior controllo della malattia. Nelle sindromi mielodisplastiche, il trattamento con Luspatercept, un nuovo meccanismo d’azione efficace contro l’anemia, ha evidenziato un miglioramento della sopravvivenza globale nei pazienti rispondenti. Questi risultati sono stati presentati al Congresso della Società Americana di Ematologia (American Society of Hematology) svolto a New Orleans. “Registriamo progressi entusiasmanti che danno uno sguardo all’incredibile ampiezza e portata delle indagini di ricerca per trovare trattamenti migliori e persino cure, specialmente per i pazienti che sono pesantemente pretrattati e che non avevano altre opzioni terapeutiche” ha dichiarato la moderatrice Stephanie Lee.

___________________

Fonte: American Society of Hematology

VERIFICATO ALLA FONTE | Guarda il protocollo di Fact checking delle notizie di Mezzopieno

 BUONE NOTIZIE CAMBIANO IL MONDO | Firma la petizione per avere più informazione positiva in giornali e telegiornali

Se trovi utile il nostro lavoro e credi nel principio del giornalismo costruttivo non-profit | sostieni Mezzopieno

[drcare4u]

Why Transfusion Independence Matters for Patients With MDS

Among patients with myelodysplastic syndromes (MDS), treatment with Reblozyl (luspatercept) resulted in at least 12 weeks of blood transfusion independence as well as a significant increase in hemoglobin, researchers have reported. The phase 3 COMMANDS study enrolled 363 adult patients with MDS of very low risk, low risk or intermediate risk who had not been treated with…

[indiamedicaltourism]

Thalassemia Treatment in India at Affordable Cost

Thalassemia is a hereditary blood condition that affects the production of hemoglobin in the body. Hemoglobin is the protein that allows red blood cells to carry oxygen throughout the body. It is an inherited condition that can be passed down from parents to their children through their DNA.  Thalassemia causes anemia in the body that causes fatigue, weakness, and shortness of breath in the body.

There are two different types of thalassemia - alpha and beta. Mild cases of thalassemia do not require any invasive treatments, but serious cases require continuous blood transfusions. About 4.4 out of every 10,000 live births around the world are affected by thalassemia. Alpha-thalassemia is more common in Southeast Asians, Middle Easterners, and individuals of African heritage. Beta-thalassemia is more common in prople of Mediterranean origin.

India has some of the best hospitals for thalassemia treatment that provide specialized care for people suffering from this condition. The symptoms of thalassemia vary depending on the type and its complexity. Common symptoms of thalassemia include fatigue, weakness, pale skin, slow growth, abdominal swelling, dark urine, trouble breathing, feeling cold, dizziness, palpitations, jaundice, and enlarged spleen.

Thalassemia can cause growth delays in children, the formation of gallstones, abnormal bone development, weakened bones, decreased fertility, as well as heart and liver complications and an increased risk to infections.

Blood transfusions are effective in managing thalassemia symptoms by producing healthy red blood cells and improving oxygen transport throughout the body. Thalassemia can be diagnosed through various tests and screenings. Blood tests are used to diagnose the condition and determine its complexity; other diagnostic tests include the Complete Blood Count (CBC), Reticulocyte Count, Iron Studies, Haemoglobin Electrophoresis, and Genetic Testing. Blood tests can also be used to test for thalassemia during pregnancy.

The standard common treatment for thalassemia is continuous blood transfusions. However, this treatment can lead to iron overload, which can damage organs such as the heart and liver. Folic acid supplements are also prescribed to help the body produce healthy blood cells. Other treatment options for thalassemia include Bone Marrow or Stem Cell Transplant, Luspatercept, Hormone Therapy, Vaccinations and Antibiotics, Thyroid Hormone Replacement, and Bisphosphonates. Bone Marrow or Stem Cell Transplant is a rare and high-risk treatment option kept for the most severe patients.

Untreated thalassemia can result in serious complications such as heart failure, liver issues, iron overload, and recurrent infections. The severity of the disease and the effectiveness of the treatment plan impact the life expectancy of people with thalassemia.

The total cost of thalassemia treatment in India includes a 15-day hospital stay, followed by 90 days of outpatient care. This includes preoperative diagnostics, the procedure itself, and treatment choices such as chelation therapy, stem cell therapy, and blood transfusion, post-operative costs, including follow-up appointments, medication, and the patient's hospital stay. The cost of each blood transfusion can be around 160 USD. Bone marrow transplant costs may vary based on the type of transplant the patient need.

Autologous bone marrow transplant: USD 16,000 - 25,000

Allogenic bone marrow transplant: USD 25,000 - 38,000

Haplo bone marrow transplant: USD 37,500 - 50,000

Unrelated bone marrow transplant: USD 60,000 - 90,000

Al Afiya Medi Tour is a leading medical tourism company in India. We offer medical tourism services such as finding the right doctor, the right hospital, and cost estimation etc. Some of the main countries are Bangladesh, South Africa, Egypt, Uganda, Zambia, Sudan, Dubai, Namibia, Iraq, Kenya, Saudi Arabia, Ethiopia, Nigeria, and so on. We provide free medical assistance for TURP surgery cost, lung cancer treatment, arthroscopic surgery, bone marrow transplant, best liver transplant hospital, brain tumor surgery, cosmetic andplastic surgery, heart surgery, kidney transplant, spine tumor surgery,lung transplant,liver transplant treatment, top knee replacement surgeons, knee replacement surgery, top shoulder replacement surgeons,  etc.

Source: https://alafiyameditour1.blogspot.com/2024/03/thalassemia-treatment-in-india-at.html

[monica-bing1920]

Luspatercept injection Reblozyl 75mg

[shesharesomething]

Bristol Myers Squibb - U.S. FDA Approves Bristol Myers Squibb’s Reblozyl® (luspatercept-aamt) as First-Line Treatment of Anemia in Adults with Lower-Risk Myelodysplastic Syndromes (MDS) Who May Require Transfusions (bms.com)

[tumimmtxpapers]

The Management of Low-Risk Myelodysplastic Syndromes-Current Standards and Recent Advances

The myelodysplastic syndromes (MDSs) are a heterogeneous group of hematologic neoplasms with varied natural histories and prognoses. Specific to this review, treatment of low-risk MDS most often focuses on improving quality of life by correcting cytopenias, as opposed to urgent disease modification to avoid acute myeloid leukemia. These treatments include transfusion support with iron chelation when necessary, growth factors including novel maturation agents such as luspatercept, lenalidomide... http://dlvr.it/SpHWtT

[medicomunicare]

Le terapie innovative per la beta-talassemia: un salto al di là della vecchia terapia chelante

Le terapie innovative per la beta-talassemia: un salto al di là della vecchia terapia chelante

Beta-talassemia: il panorama attuale Le β-talassemia sono un gruppo di malattie ematologiche ereditarie causate da più di 300 mutazioni del gene della β-globina umana, che causano una produzione bassa o assente di β-globina adulta ed un eccesso di contenuto di α-globina nelle cellule eritroidi e causano eritropoiesi inefficace e produzione bassa o assente di emoglobina adulta (HbA). Nonostante la…

[bidhuan]

FDA Setujui Terapi Pertama untuk Pasien Berkelainan Darah Langka "Beta Thalassemia"

FDA Setujui Terapi Pertama untuk Pasien Berkelainan Darah Langka “Beta Thalassemia”

farmasetika.com – Pada 8 November 2019, Badan Administrasi Obat dan Makanan AS memberikan persetujuan kepada Reblozyl (luspatercept-aamt) untuk pengobatan anemia (kekurangan sel darah merah) pada pasien dewasa dengan thalassemia beta yang memerlukan transfusi sel darah merah (red blood cell /RBC) reguler.

Persetujuan bersejarah

“Ketika pasien menerima beberapa transfusi darah, ada risiko…

[lucadezzani]

Top Pharma News of the Week - July 9th

Top Pharma News of the Week – July 9th

All The Most Important Pharmaceutical News Stories Of The Week – July 2018 – Week #2 FDA Grants Priority Review to Merck’s Supplemental Biologics License Application for KEYTRUDA® (pembrolizumab) in Combination with Chemotherapy as First-Line Treatment for Metastatic Squamous Non-Small Cell Lung Cancer

July 2, 2018

Merck, known as MSD outside the United States and Canada, has announced that the…

[medicowesome]

Luspatercept

http://dlvr.it/RJTqvs

[marcioaugustoantoniassi]

Aprovações da Anvisa: anemia, asma e psoríase em placas

Aprovações da Anvisa: anemia, asma e psoríase em placas

Luspatercepte para adultos com anemia dependente de transfusão (novo registro) – No dia 06 de dezembro, a Agência Nacional de Vigilância Sanitária (Anvisa) aprovou o registro do luspatercepete, nome comercial Reblozyl ®, produzido pela farmacêutica Bristol-Meyers Squibb. Pertencente à classe dos antianêmicos, o luspatercepte é uma nova proteína de fusão recombinante, primeira da classe que se…

[indiamedicaltourism]

Thalassemia Treatment in India at Low Cost

Thalassemia is an inherited blood disease caused by defective hemoglobin production, which causes an insufficient supply of oxygen by red blood cells. This results in anemia, which causes symptoms such as tiredness, weakness, and pale skin. This condition affects approximately 4.4 out of every 10,000 live births worldwide, with prevalence in countries like Iraq, Uzbekistan, Bangladesh, Pakistan, Nigeria, and Liberia. Thalassemia's global prevalence emphasizes its importance as a public health issue, particularly in areas with high rates of consanguineous marriage.

Types of Thalassemia

Alpha-thalassemia: It is caused by irregularities in the alpha-globin genes and can range from silent carriers to severe cases such as Alpha-Thalassemia Major.

Beta-thalassemia: It is caused by mutations in the beta-globin genes and is classified into three types: minor, intermediate, and major.

Delta-Beta Thalassemia: An uncommon subtype that affects the delta- and beta-globin genes.

Epsilon-Gamma-Delta-Beta Thalassemia: Another rare form that affects many globin genes.

Thalassemia Treatment Options

Blood transfusions: These are essential for improving healthy red blood cells.

Iron Chelation Therapy: It treats excess iron levels after transfusions.

Folic acid supplements: They promote red blood cell formation.

Bone Marrow or Stem Cell Transplant: It is used for severe situations to replace damaged blood cells.

Luspatercept: It is a new medicine that promotes red blood cell production.

Hormone therapy, vaccinations, antibiotics, thyroid hormone replacement, and bisphosphonates: These are adjunctive medicines that address specific problems.

Cost of Thalassemia Treatment in India

Thalassemia treatment in India is known for its low cost, which attracts patients from all over the world. The cost varies with the type of treatment required. Blood transfusions are required to manage thalassemia and generally cost approximately $150 each session. On the other hand, bone marrow transplants, a more advanced treatment reserved for serious conditions, can cost between $16,000 and $48,000, depending on the kind of transplant and hospital chosen.

Untreated thalassemia can cause heart failure, liver difficulties, infections, and a shorter life expectancy. Thalassemia, while difficult to treat, may be efficiently controlled with the correct medication. India's modern medical infrastructure, competent staff, and affordable care make it a perfect location for anyone seeking comprehensive thalassemia treatment. Patients with thalassemia can manage the disease with determination and hope for a better quality of life because of early diagnosis, personalized medications, and continued care.

Thalassemia treatment in India combines cost, experience, and modern technologies, making it a popular choice for patients worldwide. With top hospitals, renowned doctors, and wide-ranging therapy choices, those suffering from thalassemia may get the support and care they need to handle this difficult illness.

Al Afiya Medi Tour is a leading medical tourism company in India. We offer medical tourism services such as finding the right doctor, the right hospital, and cost estimation etc. Some of the main countries are Bangladesh, South Africa, Egypt, Uganda, Zambia, Sudan, Dubai, Namibia, Iraq, Kenya, Saudi Arabia, Ethiopia, Nigeria, and so on. We provide free medical assistance for TURP surgery cost, lung cancer treatment, blood cancer treatment, the best hospital for heart valve replacement, bone marrow transplant, best liver transplant hospital, kidney transplant, spine tumor surgery,knee replacement surgery,  etc. 

Source: https://alafiyameditour1.blogspot.com/2024/02/thalassemia-treatment-in-india-at-low.html

[awesomechrisharry]

Hemoglobinopathy Testing Services Market Projected To Experience Major Revenue Boost by 2024: Novartis, AstraZeneca, Bluebird Bio, Bristol-Myers Squibb

"The latest Hemoglobinopathy Testing Services market research report offers a comprehensive analysis of the changing trends, supply-demand scenario, high growth opportunities, and future prospects of this business sphere."

AMR has added a new report titled Hemoglobinopathy Testing Services Market with analysis provides the insights which bring marketplace clearly into the focus and thus help organizations make better decisions. Hemoglobinopathy Testing Services Market report objective is to describe market sizes of different segments and countries in recent years and to forecast to the coming seven years. Report provides strategy and advance marketing, market-entry, market extension, and other business plans by identifying the key market opportunities and prospects.

If you are an investor/shareholder in the Hemoglobinopathy Testing Services Market, the provided study will help you to understand the growth model of the Hemoglobinopathy Testing Services Industry after the impact of COVID-19.

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The Hemoglobinopathy Testing Services Market is expected to have a highly positive outlook for the next five years 2021-2024 according to a recently released. The research report is committed to giving its readers an unbiased point of view of the global Hemoglobinopathy Testing Services market. Thus, along with statistics, it includes opinions and recommendation of market experts. This allows the readers to acquire a holistic view of the global market and the segments therein. The research report includes the study of the market segments on the basis of type, application, and region. This helps in identifying segment-specific drivers, restraints, threats, and opportunities.

Top Key market Vendors Profiled in this report are:

Novartis, AstraZeneca, Bluebird Bio, Bristol-Myers Squibb, Emmaus Medical, Acceleron Pharma, HemaQuest Pharmaceuticals, Eli Lilly and Company, Celgene Corporation

Hemoglobinopathy Testing Services Market Segmentation:

By Product Type

Hydroxyurea, Glutamine, Zynteglo, Luspatercept

By Application

Sickle Cell Diseases, Thalassemia

The key questions answered in this report:

What will be the Market Size and Growth Rate in the forecast year?

What are the Key Factors driving Hemoglobinopathy Testing Services Market?

What are the Risks and Challenges in front of the market?

Who are the Key Vendors in Hemoglobinopathy Testing Services Market?

What are the Trending Factors influencing the market shares?

What are the Key Outcomes of Porter’s five forces model?

Which are the Global Opportunities for Expanding the Hemoglobinopathy Testing Services Market?

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Geographically, the Global Hemoglobinopathy Testing Services Market is designed for the following Regional Markets:

North America (USA, Canada and Mexico)

Europe (Germany, France, UK, Russia and Italy)

Asia-Pacific (China, Japan, Korea, India and Southeast Asia)

South America (Brazil, Argentina, Colombia etc.)

Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

📷

Hemoglobinopathy Testing Services Market Analysis Report Forecast 2021-2024

If you have any special requirements, please let us know and we will offer you the report as per your requirements.

Inquire or share your questions if any before the purchasing this report : http://www.acquiremarketresearch.com/enquire-before/372216

Table of Contents:

Global Hemoglobinopathy Testing Services Market Research Report 2021 – 2024

Chapter 1 Hemoglobinopathy Testing Services Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Hemoglobinopathy Testing Services Market Forecast

Read More Industry Reports @ https://marketstrategyideas.wordpress.com/

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[letrang39]

Thuốc Reblozyl 25mg – Công dụng – Liều dùng – Giá bán

Liên hệ để biết giá !

Thuốc Reblozyl 25mg là thuốc có tác dụng điều trị thiếu máu ở bệnh nhân trưởng thành nguyên nhân do bệnh beta thalassemia cần truyền máu hồng cầu (RBC) thường xuyên. Thuốc Reblozyl có chứa thành phần chính Luspatercept là một chất có tác dụng thúc đẩy sự trưởng thành hồng cầu ở giai đoạn cuối của quá trình tạo hồng cầu. Hiện nay, trên thị trường có rất nhiều loại thuốc điều trị thiếu máu. Tuy nhiên, mỗi tình trạng và bệnh nền của bệnh nhân sẽ phù hợp với những loại thuốc điều trị khác nhau.

[pharmaphorumuk]

CHMP backs BMS’ first-in-class anaemia drug Reblozyl

Bristol-Myers Squibb has moved a step closer to EMA approval of Reblozyl, a therapy for anaemia that has been billed as one of the top prospects in its $74bn buyout of Celgene.

The CHMP has recommended approval of Reblozyl (luspatercept) – a first-in-class erythroid maturation agent (EMA) originally developed by Acceleron – to treat anaemia in both the rare blood disorder beta thalassaemia and haematological cancer myelodysplastic syndrome (MDS).

Reblozyl was approved for the beta thalassaemia indication in the US last year, and for MDS last month, with the latter indication considered to be critical if it is to meet its blockbuster sales projections.

It is one of five drugs from Celgene’s pipeline tipped to top the $1 billion-a-year threshold along with myelofibrosis therapy Inrebic (fedratinib), Zeposia (ozanimod) for multiple sclerosis, and cell therapies lisocabtagene maraleucel for lymphoma and idecabtagene vicleucel for myeloma.

The EMA’s human medicines committee issued a positive opinion for Reblozyl for adults with transfusion-dependent anaemia, caused by either thalassaemia or MDS with ring sideroblasts, a form of the cancer diagnosed by red blood cells with mitochondria that form a characteristic ring around the nucleus.

While uncommon, MDS with sideroblasts is still a much larger patient population than thalassaemia. Analysts have predicted that the drug could eventually reach sales of about $2 billion, with MDS accounting for the bulk of those revenues.

There are estimated to be around 1,000 to 1,500 people with thalassemia in the US who require blood transfusions, but more than 20,000 MDS patients who might qualify for Reblozyl treatment, according to Acceleron.

Reblozyl’s role in thalassaemia could be reduced following the recent approval of bluebird bio’s gene therapy Zynteglo, which offers a one-shot therapy for the blood disorder and was approved by the EMA last year.

If the EMA goes ahead and approves Reblozyl – which usually follows endorsement by the CHMP – the drug will be the first EMA to teach the EU market.

Some patients with anaemia caused by MDS don’t respond to current therapies based on erythropoietin (EPO), according to Dr Uwe Platzbecker of Leipzig University Hospital, the lead investigator of the MEDALIST study of Reblozyl in MDS.

“If approved, the introduction of a new class of therapy in Reblozyl could provide a promising option to help relieve patients from the burden of regular transfusions to manage their disease,” he said.

BMS and Acceleron are also planning to extend the uses for Reblozyl even further, although that programme has been disrupted by the coronavirus pandemic.

They have a phase 3 trial in the pipeline that would test Reblozyl in myelofibrosis patients with anaemia requiring blood transfusions who are on JAK inhibitor treatment.

That trial – called INDEPENDENCE – is however among those affected by BMS’ decision to suspend all new clinical trial sites due to COVID-19 in March.

The post CHMP backs BMS’ first-in-class anaemia drug Reblozyl appeared first on .

from https://pharmaphorum.com/news/chmp-backs-bms-first-in-class-anaemia-drug-reblozyl/