Home » Author Archives: Catherine Skari

Author Archives: Catherine Skari

GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe

Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease (SCD) who are 12 or older. The company plans to submit the MAA by mid-2021. “Currently, there are no approved ...

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Thalassaemia Prior and Consequent to COVID-19 Pandemic: The Perspective of Thalassaemia International Federation (TIF)

  Patients with haemoglobin disorders, particularly β-thalassaemia or sickle cell disease (SCD) or combined forms, on account of their underlying disease pathology and associated (iron load mainly in the case of thalassaemia) co-morbidities, are defined as high-risk individuals, prone to develop more severe complications from SARS-COV-2. Despite the fact that ...

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IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia

  IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassaemia. The FDA had previously granted Orphan Drug Designation for IMR-687 for the treatment of ...

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Gene Therapy & Thalassaemia Version 2.3: A TIF Survey

Millions of people worldwide suffer from inherited non-malignant blood disorders like thalassaemia and sickle cell disease. The culprit: genetic defects in their blood stem cells. One of the novel therapies  for thalassaemia that has stood out recently has undoubtedly been the first and only, one-time, groundbreaking gene therapy for transfusion ...

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European Commission Approves Reblozyl for the Treatment of Transfusion-dependent β-Thalassaemia

Reblozyl’s long-awaited approval in Europe has finally arrived! Bristol Myers Squibb (BMS) & Acceleron Pharma announced today that the European Commission approved Reblozyl (luspatercept) for the treatment of Transfusion-Dependent β-#Thalassaemia (TDT) in adult patients. Reblozyl has the potential to address the ineffective erythropoiesis associated with β-thalassaemia and MDS, decrease patients’ dependence ...

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TIF Thal e-Course: Testimonials from Online Learning Thalassaemia Patients

See what patients from various countries across the world who successfully took TIF‘s Thal e-Course, the ultimate self-paced, interactive, online Εducational Course for individuals with thalassaemia and their families, have to say about their experience with the course in their own language! Well-informed patients are able to gain in-depth understanding of their ...

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TIF and Hemanext® Inc. Announce New Strategic Alliance

TIF, a worldwide organization dedicated to ensuring equal access to quality healthcare for every patient with thalassaemia and other haemoglobin disorders across the world, and Hemanext Inc., a privately held medical technology company dedicated to improving patients’ quality of life by delivering a better red blood cell (RBC) replacement therapy, ...

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FDA Grants Orphan Drug Designation to Mitapivat for Treatment of Thalassaemia

Agios Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s first-in-class pyruvate kinase-R (PKR) activator Mitapivat for the treatment of patients with NTDT α- and β-thalassaemia. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety ...

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