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Tag: orphan
FDA grants orphan drug status to NBI-77860 for treatment of congenital adrenal hyperplasia
2015-01-18 02:02:31| Biotech - Topix.net
Neurocrine Biosciences, Inc. announced today that NBI-77860, a proprietary corticotropin releasing factor 1 receptor antagonist, has been granted orphan drug status by the United States Food and Drug Administration for the treatment of congenital adrenal hyperplasia a disease that affects approximately 20,000-30,000 people in the United States. "We are very pleased that the FDA has granted NBI-77860 orphan status to treat congenital adrenal hyperplasia, a devastating disease that is a significant challenge for both clinicians and patients," said Malcolm Lloyd-Smith, Chief Regulatory Officer of Neurocrine Biosciences.
Tags: status
treatment
drug
grants
Translarna Granted Orphan Drug Designation In The U.S. And Europe For The Treatment Of Mucopolysaccharidosis I
2014-12-29 01:43:03| drugdiscoveryonline Home Page
PTC Therapeutics, Inc. (Nasdaq: PTCT) today announced that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan-drug designation (ODD) to Translarna™ (ataluren) for the treatment of patients with Mucopolysaccharidosis I (MPS I)
Product Launch - Diageo's Orphan Barrel Whiskey Distilling Co Lost Prophet 22-Year-Old
2014-12-04 14:07:00| Daily beverage news and comment - from just-drinks.com
Diageo's Orphan Barrel Whiskey Distilling Co has lined up the US launch of the fourth addition to its range of whiskeys.
Tags: product
lost
launch
barrel
RetroSense Therapeutics Granted Orphan Drug Designation For Lead Product RST-001 For Retinitis Pigmentosa
2014-11-05 05:53:53| drugdiscoveryonline Home Page
RetroSense Therapeutics, LLC, a privately-held biopharmaceutical company, announced recently that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for the Company’s lead product RST-001 for the treatment of retinitis pigmentosa (RP)
Tags: product
lead
drug
granted
Biogen Idec And Swedish Orphan Biovitrum Announce European Medicines...
2014-11-04 04:36:32| Appliances - Topix.net
CAMBRIDGE, Mass. & STOCKHOLM-- --Biogen Idec and Swedish Orphan Biovitrum AB today announced that the European Medicines Agency has validated the Marketing Authorization Application of ELOCTATM , a recombinant factor VIII Fc fusion protein product candidate for the treatment of hemophilia A. The validation of the MAA initiates the EMA's review process.
Tags: european
swedish
announce
medicines