Release Date: 29 September 2021
ALLO-715 drug which is used for the treatment of multiple myeloma has been granted orphan drug designation by the US FDA. ALLO-715, an AlloCAR T therapy targeting B-cell maturation antigen (BCMA), is a potential novel treatment for multi...
Release Date: 29 September 2021
European Medicines Agency (EMA) has granted orphan designation for bomedemstat, a lysine-specific demethylase-1 (LSD1) inhibitor, for the treatment of essential thrombocythemia (ET).
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Release Date: 28 September 2021
US FDA has granted Orphan Drug Designation of CycloSamandreg; for the treatment of patients diagnosed with osteosarcoma. CycloSam drug candidate utilizes both a more efficient method of isotope manufacturing and an advanced chelation tec...
Release Date: 28 September 2021
US FDA has granted orphan drug designation to CFT7455 for the treatment of multiple myeloma.CFT7455 is an orally bioavailable MonoDACandtrade; degrader targeting IKZF1/3 for the treatment of multiple myeloma and non-Hodgkin’s lymph...
Release Date: 27 September 2021
US FDA granted the orphan designation to NNZ-2591 for treating Prader-Willi, a neurodevelopmental disorder. Neuren Pharmaceuticals outcome of the trial using a mouse model of Prader-Willi syndrome, which saw treatment for six weeks normalize fat mass, insulin levels...
Release Date: 27 September 2021
European Commission has granted TSHA-102, an AAV9-based gene replacement therapy to treat Rett syndrome. TSHA-102 delivers the MECP2 gene, which includes the novel miRNA-Responsive Auto-Regulatory Element (miRARE), to regulate MECP2 expr...
Release Date: 26 September 2021
US FDA has granted orphan drug designation to EPX-200 drug for the treatment in Lennox-Gastaut Syndrome. It is a rare epileptic encephalopathy presenting in childhood with intractable seizures. Around 48,000 children and adults in the U....
Release Date: 26 September 2021
US FDA has granted Orphan Drug designation to VS-01 to treat hyperammonemia in inborn errors of metabolism. VS-01 is a liposomal-based intraperitoneal fluid which acts as a clearance enhancer of toxic metabolites from the body, in partic...
Release Date: 25 September 2021
US FDA has allowed LP-184 Orphan Drug Designation for the treatment of pancreatic cancer. LP-184 is a small molecule drug candidate and next generation alkylating agent that specially damages DNA in cancer cells that over-express certain...
Release Date: 25 September 2021
US FDA has granted orphan drug designation to RLF-100 (aviptadil) for the treatment of Sarcoidosis. Sarcoidosis is a rare disease in which the inflammatory process includes the alveoli (air sacs), small bronchi, and small blood vessels. ...
Release Date: 24 September 2021
US FDA has allowed Paratek Pharmaceuticals the orphan drug designation for NUZYRAandreg; (omadacycline) for the treatment of Nontuberculous Mycobacteria (NTM). NUZYRAandreg; (omadacycline) is once-daily antibiotic with both oral and intr...
Release Date: 24 September 2021
US FDA has granted Aerami Therapeutics orphan drug designation for Imatinib for the treatment of pulmonary arterial hypertension (PAH). Pulmonary Arterial Hypertension (PAH) is affecting almost 68,000 patients worldwide with limited trea...
Release Date: 23 September 2021
PharmaTher Holdings Ltd. got permission from orphan drug designation (ODD) for ketamine in the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. Ketamine has the potential to effectively proliferation ...
Release Date: 23 September 2021
European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. Akouos, Inc. a precision g...
Release Date: 22 September 2021
US FDA has granted orphan drug designation to Ketamine for the treatment of amyotrophic lateral sclerosis (ALS). Ketamine is a type of psychedelic or dissociative compound that blocks sensory perception, and is used mainly for starting a...