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…
Read More » -
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…
Read More » -
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…
Read More » -
FDA Approves New Formulation for Ferriprox (Deferiprone) Twice-a-Day Tablets
Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., today announced that the U.S. Food and Drug Administration (FDA) has approved Ferriprox® (deferiprone) twice-a-day tablets for the treatment…
Read More » -
FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD
CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX-001, an investigational, autologous, Gene Editing hematopoietic stem cell therapy, for the treatment of severe Transfusion-Dependent β-Thalassaemia (TDT) and Sickle…
Read More » -
Reblozyl (luspatercept-aamt) Receives Positive CHMP Opinion for the Treatment of Adults with β-Thalassaemia
On 30 April 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product REBLOZYL, intended for the treatment of adults…
Read More » -
AABB’s Develops Toolkit with Information Regarding Blood Donation & the COVID-19 Outbreak
As the new coronavirus continues to spread affecting an increasing number of countries around the globe and in response to FDA’s Important Information for Blood Establishments Regarding the Novel Coronavirus…
Read More » -
FT-4202 Granted FDA Fast Track, Rare Pediatric Disease Designations for SCD
FT-4202, a potential disease-modifying oral treatment for sickle cell disease being developed by Forma Therapeutics, has been given fast track and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). A fast track status…
Read More » -
FDA grants Orphan Drug Designation for ARU-1801 for the Treatment of Sickle Cell Disease & β-thalassaemia
An investigational gene therapy for Sickle Cell Disease (SCD) and β-thalassaemia, known as ARU-1801, has been given Orphan Drug Designation an orphan drug by the U.S. Food and Drug Administration (FDA), a…
Read More » -
Bluebird bio announces 1st commercial launch of ZYNTEGLO™in Germany
Bluebird bio, Inc. announced earlier today the launch of ZYNTEGLO™ in Germany, the company’s one-time gene therapy for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do…
Read More »