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BioLineRx Receives Orphan Drug Designation for Motixafortide for Pancreatic Cancer in Europe
americanpharmaceuticalreview
January 19, 2020
BioLineRx announced the European Commission (EC) has granted Orphan Drug Designation to its lead oncology candidate, Motixafortide (BL-8040), for the treatment of pancreatic cancer, based on a positive opinion from the Committee for ...
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Acute myeloid leukemia treatment receives FDA Orphan Drug Designation
europeanpharmaceuticalreview
January 09, 2020
Orphan drug designation has been approved by the FDA for PRGN-3006 UltraCAR-T™ for patients with acute myeloid leukemia (AML).
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FDA Grants Avatrombopag Orphan Drug Designation
americanpharmaceuticalreview
December 24, 2019
Dova Pharmaceuticals has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for avatrombopag for the potential treament of Chemotherapy-Induced Thrombocytopenia (CIT).
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EC grants orphan drug status to Dicerna’s DCR-A1AT to treat congenital A1AT deficiency
pharmaceutical-business-review
December 20, 2019
Dicerna Pharmaceuticals has secured orphan drug designation from the European Commission (EC) for its DCR-A1AT to treat congenital alpha-1 antitrypsin (A1AT) deficiency.
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FDA grants Orphan Drug Designation to Epidermolysis Bullosa treatment
europeanpharmaceuticalreview
December 10, 2019
The US Food and Drug Administration has awarded APR-TD011 Orphan Drug Designation, for the treatment of a rare disease.
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Glenmark receives Orphan Drug Designation for GBR 1342
biospectrumasia
September 18, 2019
Derived from the company’s proprietary BEAT® (Bispecific Engagement by Antibodies based on the T cell receptor) technology, GBR 1342 is being investigated for the treatment of multiple myeloma.
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anti-BCMA autologous CAR-T cells (CT053) receives US FDA orphan drug designation
biospectrumasia
September 03, 2019
Under the Orphan Drug Act, the CT053 anti-BCMA product would be eligible for certain benefits including seven years of market exclusivity in the United States following marketing approval by the FDA.
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Neuropore Receives Orphan Drug Designation for ALS Treatment
americanpharmaceuticalreview
August 15, 2019
Neuropore Therapies has received orphan drug designation for NPT520-34 for the treatment of amyotrophic lateral sclerosis or ALS. The orphan drug designation will facilitate the development of NPT520-34 in the treatment of this debilitating medical condit
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Dynacure Receives Orphan Drug Designation for DYN101
americanpharmaceuticalreview
August 12, 2019
Dynacure announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for ...
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FDA grants orphan drug designation to Stoke Therapeutics’ STK-001
pharmaceutical-technology
August 08, 2019
The US Food and Drug Administration (FDA) has granted orphan drug designation to Stoke Therapeutics’ antisense oligonucleotide, STK-001, a new therapy for severe and progressive genetic epilepsy Dravet syndrome.