Innovative Therapies

Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU
NOVITÀ

Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU

CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are jointly…
IMR-687 Receives FDA Fast Track & Rare Pediatric Disease Designations for the Treatment of β-Τhalassaemia
Clinical News

IMR-687 Receives FDA Fast Track & Rare Pediatric Disease Designations for the Treatment of β-Τhalassaemia

IMARA Inc., a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of haemoglobin, announced the U.S. Food and Drug…
TIF Publishes its Annual Report for 2019
NOVITÀ

TIF Publishes its Annual Report for 2019

This Annual Report showcases the Federation’s intensive efforts that took place in 2019 with the aim to ensure equal access to quality healthcare for every patient with thalassaemia and haemoglobin…
Novartis sickle cell drug Adakveo put on path to EU approval
Clinical News

Novartis sickle cell drug Adakveo put on path to EU approval

Novartis announced last Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization of Adakveo® (crizanlizumab)…
TIF.ACCESS: A Global TIF Initiative for the Promotion of the Availability & Accessibility of Innovative Therapies
NOVITÀ

TIF.ACCESS: A Global TIF Initiative for the Promotion of the Availability & Accessibility of Innovative Therapies

The swift scientific progress made in the field of thalassaemia research has gained momentum with the market authorisations of both the Zynteglo Gene Therapy by the European Medicines Agency (EMA)…
GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Clinical News

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…
Advances in the Care & Cure of Thalassaemia: New Hopes & Challenges
NOVITÀ

Advances in the Care & Cure of Thalassaemia: New Hopes & Challenges

TIF, in collaboration with Thalassemics India and Thalassemia Patients Advovacy Group – India, conducted on June 12, 2020 a Webinar on the novel treatments emerging for thalassaemia and the hopes…
IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia
Clinical News

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

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

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…
Gene Therapy & Thalassaemia: A TIF Survey
Surveys

Gene Therapy & Thalassaemia: 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 …
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