Anemias

FibroGen Announces Outcome of FDA Advisory Committee Review of Roxadustat for Treatment of Anemia of Chronic Kidney Disease

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星期四, 七月 15, 2021
Enzymes, Chemical compounds, Acetic acids, Organic compounds, Isoquinolines, Roxadustat, Hypoxia-inducible factor, Anemias, Myelodysplastic syndrome, HIF prolyl-hydroxylase inhibitor, Procollagen-proline dioxygenase, Chronic kidney disease

Roxadustat, an oral small molecule hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor, is the first HIF-PH inhibitor accepted by the FDA for review for the treatment of anemia of CKD.

Key Points: 
  • Roxadustat, an oral small molecule hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor, is the first HIF-PH inhibitor accepted by the FDA for review for the treatment of anemia of CKD.
  • Roxadustat is also in clinical development for anemia associated with myelodysplastic syndromes (MDS) and for chemotherapy-induced anemia (CIA).
  • The Company is currently developing and commercializing roxadustat, an oral small molecule inhibitor of HIF prolyl hydroxylase activity, for anemia of chronic kidney disease (CKD).
  • Roxadustat is also in clinical development for anemia associated with myelodysplastic syndromes (MDS) and for chemotherapy-induced anemia (CIA).

CAF President Peter Chieco Selected as Finalist for Lifetime Achievement Award by Invest in Others Charitable Foundation

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星期四, 七月 15, 2021
Anemias, Cooley's Anemia Foundation, CAF

NEW YORK, July 15, 2021 /PRNewswire-PRWeb/ -- Cooley's Anemia Foundation congratulates Peter Chieco, CAF Volunteer National President, on being selected as one of three national finalists for the Lifetime Achievement Award as part of the 15th Annual Invest in Others Awards.

Key Points: 
  • NEW YORK, July 15, 2021 /PRNewswire-PRWeb/ -- Cooley's Anemia Foundation congratulates Peter Chieco, CAF Volunteer National President, on being selected as one of three national finalists for the Lifetime Achievement Award as part of the 15th Annual Invest in Others Awards.
  • Mr. Chieco's decades of work on behalf of the Cooley's Anemia Foundation has gained him this unique honor, and as a result, CAF will receive a $20,000 donation from the Invest in Others Charitable Foundation.
  • Notably, Mr. Chieco developed CAF's first endowment campaign, which provided the Foundation with financial stability during the COVID pandemic.
  • CAF National Executive Director Craig Butler commented, "The Cooley's Anemia Foundation is thrilled that Invest in Others Charitable Foundation has recognized the extraordinary work and dedication of Peter Chieco, our National President and a long-time member of the CAF Board of Directors.

Rockwell Medical, Inc. Files Pre-IND Meeting Request with FDA for its Proposed Clinical Trial of FPC as a Treatment for Iron Deficiency Anemia in Patients Receiving Home Infusion

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星期一, 六月 28, 2021
Medical specialties, Blood, Clinical medicine, Intravenous fluids, Anemias, Mineral deficiencies, Intensive care medicine, Parenteral nutrition, Iron deficiency, Rockwell Medical

Many patient groups requiring home infusion therapies suffer from chronic diseases that are associated with a high incidence of iron deficiency and anemia.

Key Points: 
  • Many patient groups requiring home infusion therapies suffer from chronic diseases that are associated with a high incidence of iron deficiency and anemia.
  • For example, it is estimated that 40%-55% of all home parenteral nutrition patients are iron deficient.
  • We expect to finalize our Phase 2 clinical study design and protocol with the advice and guidance of the FDA.
  • Rockwell Medical expressly disclaims any obligation to update our forward-looking statements, except as may be required by law.

Agios Submits Marketing Authorisation Application to European Medicines Agency for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency

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星期一, 六月 28, 2021
Blood, Medical specialties, Hematopathology, Anemias, Transfusion medicine, Haemolytic anaemias, Pyruvate kinase, Hemolysis, Hemolytic anemia, Kinase, Congenital hemolytic anemia, Autoimmune hemolytic anemia

A full analysis of these data including patient-reported outcomes (PRO) was recently presented at theEuropean Hematology Association (EHA) Virtual Congress.

Key Points: 
  • A full analysis of these data including patient-reported outcomes (PRO) was recently presented at theEuropean Hematology Association (EHA) Virtual Congress.
  • An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.
  • Pyruvate kinase (PK) deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells.
  • The companys most advanced drug candidate is a first-in-class pyruvate kinase R (PKR) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias.

Astellas Receives Positive CHMP Opinion for EVRENZO™ (roxadustat) for Adult Patients with Symptomatic Anemia of Chronic Kidney Disease

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星期五, 六月 25, 2021
Enzymes, Chemical compounds, Acetic acids, Isoquinolines, Roxadustat, Anemias, Myelodysplastic syndrome, Chronic kidney disease, Procollagen-proline dioxygenase, HIF prolyl-hydroxylase inhibitor, Molidustat

Todays positive CHMP opinion marks a significant step in providing patients with a new and important treatment option for anemia associated with CKD, regardless of dialysis status, saidBernhardt G. Zeiher, M.D., Chief Medical Officer, Astellas.

Key Points: 
  • Todays positive CHMP opinion marks a significant step in providing patients with a new and important treatment option for anemia associated with CKD, regardless of dialysis status, saidBernhardt G. Zeiher, M.D., Chief Medical Officer, Astellas.
  • We look forward to the review and assessment by the European Commission which, if positive, would make roxadustat the first HIF-PH inhibitor approved in Europe to treat symptomatic anemia in adult patients with CKD.
  • Roxadustat is also in Phase 3 clinical development for anemia associated with myelodysplastic syndromes (MDS) and Phase 2 for chemotherapy-induced anemia (CIA).
  • The Company is currently developing and commercializing roxadustat, an oral small molecule inhibitor of HIF prolyl hydroxylase activity, for anemia associated with chronic kidney disease (CKD).

Agios Launches myAgios® Patient Support Services for Pyruvate Kinase Deficiency

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星期二, 六月 22, 2021
Blood, Medical specialties, Hematopathology, Branches of biology, Inborn errors of carbohydrate metabolism, Pyruvate kinase, Anemias, Haemolytic anaemias, Hemolysis, Kinase, Hemolytic anemia, Congenital hemolytic anemia

CAMBRIDGE, Mass., June 22, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced the launch of myAgios patient support services for people living with pyruvate kinase (PK) deficiency and their caregivers.

Key Points: 
  • CAMBRIDGE, Mass., June 22, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced the launch of myAgios patient support services for people living with pyruvate kinase (PK) deficiency and their caregivers.
  • Pyruvate kinase deficiency is a lifelong hemolytic anemia with no disease-modifying treatments currently available to patients.
  • Patients and caregivers interested in obtaining more detailed content about PK deficiency can enroll in the patient support program at www.myagios.com/patient/pkd-enroll .
  • Pyruvate kinase (PK) deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells.

Agios Submits New Drug Application to FDA for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency

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星期一, 六月 21, 2021
Blood, Medical specialties, Hematopathology, Hematology, Inborn errors of carbohydrate metabolism, Anemias, Pyruvate kinase, Haemolytic anaemias, Transfusion medicine, Hemolysis, Hemolytic anemia, Kinase

An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.

Key Points: 
  • An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.
  • The company remains on track to submit a marketing authorization application (MAA) in the EU in mid-2021 for mitapivat in adults with PK deficiency.
  • Pyruvate kinase (PK) deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells.
  • The companys most advanced drug candidate is a first-in-class pyruvate kinase R (PKR) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias.

European Hematology Association - Above and BEYOND: Luspatercept is Efficacious and Well-Tolerated in Patients with Non-Transfusion-Dependent β-Thalassemia

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星期五, 六月 11, 2021
Medical specialties, Anemias, Clinical medicine, Blood, Transfusion medicine, Luspatercept, Thalassemia, Beta thalassemia, Transfusion-dependent anemia

Despite their transfusion-independence, patients with NTD -thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life.

Key Points: 
  • Despite their transfusion-independence, patients with NTD -thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life.
  • There are currently no approved therapies for the management of anemia in NTD -thalassemia.
  • Luspatercept is a drug that is approved for the treatment of anemia in transfusion-dependent -thalassemia and may also be beneficial for patients with NTD -thalassemia.
  • In conclusion, luspatercept is efficacious and safe for treating patients with NTD -thalassemia.

European Hematology Association - Above and BEYOND: Luspatercept is Efficacious and Well-Tolerated in Patients with Non-Transfusion-Dependent β-Thalassemia

Retrieved on: 
星期五, 六月 11, 2021
Medical specialties, Anemias, Clinical medicine, Blood, Transfusion medicine, Luspatercept, Thalassemia, Beta thalassemia, Transfusion-dependent anemia

Despite their transfusion-independence, patients with NTD -thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life.

Key Points: 
  • Despite their transfusion-independence, patients with NTD -thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life.
  • There are currently no approved therapies for the management of anemia in NTD -thalassemia.
  • Luspatercept is a drug that is approved for the treatment of anemia in transfusion-dependent -thalassemia and may also be beneficial for patients with NTD -thalassemia.
  • In conclusion, luspatercept is efficacious and safe for treating patients with NTD -thalassemia.

Global Sickle Cell Anemia Market Spotlight 2021: Global Blood Therapeutics Leads Industry Sponsors - ResearchAndMarkets.com

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星期二, 六月 8, 2021
Health, Biotechnology, Blood, Medical specialties, Branches of biology, Hemoglobins, Hematopathology, Health in Africa, Anemias, Transfusion medicine, Sickle cell disease, Fetal hemoglobin, Hemoglobin C, Sickle cell trait

Using an estimated global prevalence of 0.04%, the publisher approximated that there were 3.3 million prevalent cases of sickle cell anemia worldwide in 2019.

Key Points: 
  • Using an estimated global prevalence of 0.04%, the publisher approximated that there were 3.3 million prevalent cases of sickle cell anemia worldwide in 2019.
  • The approved drugs in the sickle cell anemia space target selectins, fetal hemoglobin, nicotinamide adenine dinucleotide, and hemoglobin.
  • Global Blood Therapeutics has the highest number of completed clinical trials for sickle cell anemia, with 10 trials.
  • Global Blood Therapeutics leads industry sponsors with the highest overall number of clinical trials for sickle cell anemia, followed by Novartis