Release Date: 14 September 2021
Fidia Farmaceutici receives Orphan Drug Designation from US FDA for ONCOFIDandreg;-P for the treatment of malignant mesothelioma (MM). ONCOFIDandreg;-P is an advanced anti-cancer drug, a conjugate of paclitaxel (taxol) w...
Release Date: 14 September 2021
Ultragenyx Pharmaceutical Inc. receives US FDA and European Commission for UX053 for the treatment of Glycogen Storage Disease Type III (GSDIII). UX053 is an investigational mRNA-based biologic therapy encoding full-length, glycogen debr...
Release Date: 13 September 2021
(Zydus Cadila), a healthcare Ltd has received Orphan Drug Designation from the EU regulator, European Medicines Agency (EMA), for Saroglitazar Mg to treat Primary Biliary Cholangitis (PBC). PBC is a liver disease, caused due to progressi...
Release Date: 13 September 2021
US FDA grants Orphan Drug Designation for pridopidine for the treatment of Amyotrophic Lateral Sclerosis (ALS). Prilenia Therapeutics B.V, a clinical stage biotech company develops pridopidine, a highly selective S1R agonist investigatio...
Release Date: 12 September 2021
US FDA has granted Orphan Drug Designation to TST001 for the treatment of gastric cancer or gastroesophageal junction (GC/GEJ). Gastric cancer is a rare disease in US but has a higher incidence in Asian countries such as China and Japan....
Release Date: 12 September 2021
Zogenix, a global biopharmaceutical company announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation (ODD) to FINTEPLAandreg; (fenfluramine) oral solution to treat Dravet syndrome. Dra...
Release Date: 11 September 2021
Coya Therapeutics, Inc. receives the orphan drug designation from US FDA for ALS001, an autologous, expanded Treg cell therapy for the treatment of amyotrophic lateral sclerosis (ALS). ALS is a progressive neurodegenerative disease initi...
Release Date: 11 September 2021
US FDA has granted Orphan Drug Designation for VO659 for the treatment of patients with Huntington’s disease (HD). VO659 is developed to suppress mutant proteins with the intent of reducing or halting the progression of disease. Pr...
Release Date: 10 September 2021
Bioheng Biotech Co., Ltd. received Orphan Drug Designation from the U.S FDA for the treatment of T-cell acute lymphoblastic leukemia (T-ALL). CTD401 is a universal CAR-T (UCAR-T) cell therapy product developed for the treatment of patien...
Release Date: 10 September 2021
Thetis Pharmaceuticals receives orphan drug designation (ODD) from US FD to TP-317 for the treatment of Pancreatic Cancer. Pancreatic cancer is a lethal disease that accounts for 3.2% of cancer cases in the U.S. (57,600 in 2020) but cont...
Release Date: 09 September 2021
U.S. FDA has granted Orphan Drug Designation status to etrasimod, a highly selective, once-daily, oral sphingosine 1-phosphate (S1P) receptor modulator, for the treatment of eosinophilic esophagitis (EOE).Etrasimod (APD334) is discovered...
Release Date: 09 September 2021
Jasper Therapeutics, Inc., a biotechnology earns orphan drug designation from US FDA to JSP191, a monoclonal antibody targeting the CD117 (stem cell factor) receptor, for conditioning treatment prior to hematopoietic stem cell transplant...
Release Date: 08 September 2021
US FDA has granted Orphan Drug Designation to DCCR (diazoxide choline) Extended-Release tablets to treat Glycogen Storage Disease Type 1a (GSD 1a), or von Gierke disease. GSD 1a is a disease marked by the body’s inability to metabolize glycogen into glucose, l...
Release Date: 08 September 2021
Spinogenix, Inc. a pharmaceutical company has been granted Orphan Drug Designation for SPG302 in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) by the U.S FDA. SPG302 is an orally bioavailable, blood-brain barrier penetratin...
Release Date: 07 September 2021
European Medicines Agency (EMA) has allowed orphan drug designation for elamipretide for the treatment of Barth syndrome. Barth syndrome is an extreme-rare genetic condition characterized by cardiac abnormalities which leads to reduced l...