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FDA grants Orphan Drug Designation to DYN101 treatment europeanpharmaceuticalreview
August 13, 2019
A treatment for Centronuclear Myopathies has received Orphan Drug Designation from the FDA and will begin Phase I/II trials.
Mustang Bio’s AML Treatment Receives Orphan Drug Designation contractpharma
July 31, 2019
MB-102 (CD123 CAR T) evaluated to treat AML and BPDCN.
EMA grants AR-501 with Orphan Drug Designation europeanpharmaceuticalreview
July 23, 2019
Orphan Drug Designation has been given to AR-501 for the treatment of lung infection in patients with cystic fibrosis.
FDA grants Orphan Drug Designation to Imfinzi for SCLC europeanpharmaceuticalreview
July 16, 2019
The FDA has announced Orphan Drug Designation for Imfinzi (durvalumab) to treat small cell lung cancer in combination with other therapies.
Aridis Pharmaceuticals Receives Orphan Drug Designation for AR-501 americanpharmaceuticalreview
June 28, 2019
Aridis Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AR-501, the Company's inhaled formulation of gallium citrate for treatment of lung infection in patients with cystic fibrosis.
FDA grants Orphan Drug Designation for narcolepsy treatment europeanpharmaceuticalreview
June 27, 2019
The FDA has given the XWL-008 compound Orphan Drug Designation for the treatment of narcolepsy.
Implandata Successfully Gains CE Mark for its EYEMATE System for an Orphan Eye Indication b3cnewswire
May 05, 2019
Implandata Successfully Gains CE Mark for its EYEMATE System for an Orphan Eye Indication.
EC grants orphan drug designation for Gilteritinib for the treatment of AML europeanpharmaceuticalreview
April 29, 2019
The European Commission has issued Orphan Designation to gilteritinib for the treatment of patients with acute myeloid leukaemia…
BridgIT, a new tool for orphan and novel enzyme reactions worldpharmanews
March 27, 2019
Effective protein engineering can give us control over the generated products inside a cell.
FDA Approves First Treatment for Rare Autoimmune Disorder contractpharma
November 30, 2018
Catalyst Pharmaceuticals, Inc. received approval from the FDA for Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves

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