Clinical News
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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…
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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…
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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…
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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…
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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…
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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…
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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…
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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…
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Deferasirox Accord: A new generic of Exjade recommended for approval by the EMA
The Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency‘ (EMA) committee responsible for human medicines, has recently adopted a positive opinion recommending the granting of a…
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Deferasirox Mylan: A new generic drug for the treatment of iron overload in β-thalassaemia major obtained approval by the EMA
The European Medicines Agency’s (EMA) has recently granted marketing authorisation for the medicinal product ”Deferasirox Mylan”, intended for the treatment of chronic iron overload due to blood transfusions in patients with…
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