Bayer HealthCare Pharmaceuticals, a subsidiary of Bayer, has obtained two separate orphan drug designations from the US Food and Drug Administration's (FDA) Office of Orphan Products Development for its investigational, oral medication ...
Tags: Drug Designations, Medicine
The European Medicines Agency (EMA) has granted an orphan drug status to US-based clinical-stage biotechnology company GlycoMimetics' GMI-1070 (rivipansel sodium), designed to treat vaso-occlusive crisis (VOC) in patients with sickle cell ...
The European Medicines Agency (EMA) has granted an orphan drug status to US-based clinical-stage biotechnology company GlycoMimetics’ GMI-1070 (rivipansel sodium), designed to treat vaso-occlusive crisis (VOC) in patients with sickle ...
Tags: Orphan Drug, Cell Drug
RNAi therapeutics company Alnylam Pharmaceuticals has obtained additional orphan drug designation from US FDA for a RNAi therapeutic, ALN-AT3, to treat hemophilia A. With the additional approval, orphan drug designation for GalNAc ...
Tags: Alnylam, Orphan Drug
The European Commission has granted orphan drug designation to BrainStorm Cell Therapeutics’ stem cell therapy NurOwn to treat Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease. NurOwn consists of autologous ...
Tags: Orphan Drug, Medicine
La Jolla Pharmaceutical Company has reported a comprehensive net loss attributable to common stockholders of $3.74m, or $0.14 per share, for the second quarter ended on 30 June 2013 compared with $7.77m, or $0.67 per share in 2012. ...
Tags: La Jolla Pharmaceutical, Net Loss
Biopharmaceutical development company Chelsea Therapeutics has announced the US FDA acceptance of Northera (droxidopa) new drug application (NDA) resubmission to treat symptomatic neurogenic orthostatic hypotension (NOH). The company is ...
The US Patent and Trademark Office (USPTO) has issued a notice of patent allowance for DelMar Pharmaceuticals’ first-in-class small-molecule chemotherapeutic VAL-083. The patent covers the methods for synthesizing VAL-083, which is ...
The Japanese Ministry of Health, Labour and Welfare (MHLW) has cleared Roche's Avastin (bevacizumab) as a combination therapy and monotherapy to treat the aggressive form of brain cancer glioblastoma. The first new medicine that is ...
Tags: Glioblastoma Therapy, Medicine
Stemline Therapeutics' SL-401 has won orphan drug designation from the Food and Drug Administration (FDA) for the treatment of aggressive hematologic malignancy blastic plasmacytoid dendritic cell neoplasm (BPDCN). With an orphan drug ...
Tags: Stemline Therapeutic, Orphan Drug
Clinical-stage pharmaceutical company Cempra has received a notification of patent allowance from the US Patent and Trademark Office (USPTO) for taksta loading dose formulation. With the possibility for extension, the patent entitled ...
Basilea Pharmaceutica has obtained FDA orphan drug status to isavuconazole, an investigational intravenous and oral broad-spectrum antifungal, for the treatment of invasive aspergillosis. Isavuconazole is being investigated in phase 3 ...
The US Food and Drug Administration (FDA) has cleared Omeros' investigational new drug application (IND) for OMS824 in Huntington's disease. OMS824 is developed to selectively inhibit phosphodiesterase 10 enzyme that is expressed in areas ...
Tags: Omeros'IND, Medicine
The US Food and Drug Administration (FDA) has granted orphan drug exclusivity to Hyperion's Ravicti (glycerol phenylbutyrate) liquid. Hyperion Therapeutics president and CEO Donald Santel said that the orphan drug exclusivity further ...
Tags: Hyperion, Ravicti Liquid
Teva Pharmaceutical and Xenon Pharmaceuticals have announced the FDA orphan drug designation for analgesic XEN402 being developed to treat pain associated with erythromelalgia (EM). The novel chemical entity XEN402 is designed to inhibit ...
Tags: Pharmaceutical, FDA, Orphan Drug