Latest Orphan drug Stories
Aeterna Zentaris to continue ongoing Phase 3 trial in multiple myeloma QUEBEC CITY, May 7, 2012 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced that it and Keryx Biopharmaceuticals, Inc. ("Keryx") (NASDAQ: KERX) have agreed to terminate their license agreement with respect to perifosine, as a result of which Aeterna Zentaris has regained in full the North American rights to its Akt inhibitor anticancer compound, perifosine, in all...
SAN DIEGO, April 9, 2012 /PRNewswire/ -- ADVENTRX Pharmaceuticals, Inc. (NYSE Amex: ANX) today announced the establishment of a Regulatory Advisory Board (RAB). The RAB will provide drug development and regulatory affairs guidance to Company management in its interactions with the U.S. Food and Drug Administration (FDA). Frank J. Sasinowski, M.S., M.P.H., JD, who is rendering legal advice to Company management, and in that capacity also will advise the RAB, is a Director at Hyman,...
NOVATO, Calif., March 28, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the European Medicines Agency (EMA) has granted orphan drug designations for sialic acid for the treatment of hereditary inclusion body myopathy (HIBM) and recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type 7 (MPS 7). Orphan designation in the European...
THE WOODLANDS, Texas, March 20, 2012 /PRNewswire/ -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), announced today that the U.S. Food and Drug Administration (FDA) has granted its request for Orphan Drug designation for telotristat etiprate (LX1032) for the treatment of carcinoid syndrome. Carcinoid syndrome is a chronic condition caused by neuroendocrine tumors that usually originate from the gastrointestinal tract and is characterized by severe diarrhea and flushing episodes with...
NOVATO, Calif., March 8, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the Phase 1 study of UX001 for hereditary inclusion body myopathy (HIBM) has been completed. UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency. UX001 is the...
NOVATO, Calif., Feb. 28, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the...
How can insurers justify spending hundreds of thousands of dollars per patient per year on "orphan drugs" – extremely expensive medications for rare conditions that are mostly chronic and life-threatening -- when this money could provide greater overall health benefit if spread out among many other patients? Those spending decisions reflect the "rule of rescue," the value that our society places on saving lives in immediate danger at any expense. But the broad application of the rule of...
Jack Clapper is an expert asbestos and mesothelioma attorney and founder of one of the first mesothelioma law firms in California, Clapper Patti Schweizer & Mason (CPSM), which specializes in representing those injured by asbestos. Having been in the field for over three decades, Clapper and his firm not only understands the complexities of asbestos litigation and bankruptcy trust claims, but also the urgent need to provide the most up-to-date information regarding treatments for...
BLAINVILLE, QC, Nov. 29, 2011 /PRNewswire/ - Médunik Canada, a pharmaceutical company dedicated to bridging the gap by helping Canadians with rare diseases have access to orphan drugs currently unavailable in Canada, has announced the conclusion of a strategic collaboration agreement with QOL Medical LLC. Under this agreement, Médunik Canada receives the exclusive Canadian rights to make available and distribute to patients and physicians two important therapies for...
Released at Major Conference, Study Catalogues Flexibility in Orphan Drugs Approved Since 1983 WASHINGTON, Oct. 11, 2011 /PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) today released a landmark report documenting flexibility in the Food and Drug Administration (FDA) review of potential treatments for patients with rare diseases. (Logo: http://photos.prnewswire.com/prnh/20110719/DC37656LOGO-b) Released at the U.S. Conference on Rare Diseases and Orphan...
