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Last updated on April 19, 2014 at 13:20 EDT

Latest Orphan drug Stories

2013-12-03 12:26:09

CARLSBAD, Calif., Dec. 3, 2013 /PRNewswire/ -- Agility-PSR - the partnership between Agility Clinical, Inc., a Carlsbad-based specialty consulting and clinical research organization (CRO), and PSR Orphan Experts, a European orphan drug specialty consulting and clinical research company - has been awarded "Best Contract Research Organization" in the 2013 Rare and Orphan Advocacy and Research (ROAR) Awards. The ROAR Awards were announced during the 4(th) Annual World Orphan Drug...

2013-12-02 08:30:54

DURHAM, N.C., Dec. 2, 2013 /PRNewswire/ -- Scioderm, Inc. announced the appointment of John Crowley and William Aliski to the company's Board of Directors. As Chairman & CEO of NASDAQ listed Amicus Therapeutics, John brings a significant rare and orphan disease experience to Scioderm, in addition to his in depth transactional and public sector market experience. His personal drive, commitment and overall determination to find cures for rare diseases such as Pompe and Fabry...

2013-11-15 04:21:02

- Orphan Drug Designation Granted for Epidiolex in Dravet syndrome by the FDA - LONDON, Nov. 15, 2013 /PRNewswire/ -- GW Pharmaceuticals plc (AIM: GWP, Nasdaq: GWPH, "GW") announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Epidiolex®, our product candidate that contains plant-derived Cannabidiol (CBD) as its active ingredient, for use in treating children with Dravet syndrome, a rare and severe form of infantile-onset, genetic,...

2013-11-13 08:30:23

High Profiles of Biotechs Specializing in Rare Diseases Has Pushed Up the Price Per Dollar of Future Sales Above Those in Other Fields, According to Findings from Decision Resources BURLINGTON, Mass., Nov. 13, 2013 /PRNewswire/ -- Decision Resources, one of the world's leading research and advisory firms focusing on pharmaceutical and healthcare issues, finds that ultra-orphan drug specialists are among the lowest value for money for pharmaceutical companies seeking to boost their...

2013-11-11 08:28:05

VANCOUVER, British Columbia and MENLO PARK, Calif., Nov. 11, 2013 /PRNewswire/ -- Dear Shareholders and Friends of DelMar Pharmaceuticals: I am pleased to provide you with an update as of the quarter ended September 30, 2013 and a summary of progress toward our goals for the year. Our Financials Are on BudgetAs of September 30, 2013 we have cash and equivalents of approximately $5.2 million. Our working capital balance is in line with our expectations. Based on our operating...

2013-11-07 08:31:45

-- Obtains Additional $17.4 Million in Cash in 4Q 2013 -- SEATTLE, Wash., Nov. 7, 2013 /PRNewswire/ -- -- Received $17.4 million in cash in October 2013 -- Total operating expenses in 3Q 2013 were $13.6 million including $3.2 million in non-cash expenses compared to $13.3 million including non-cash expenses of $2.1 million in 2Q 2013 -- U.S. and European regulators accepted Omeros' OMS302 marketing applications for review -- OMS824 and OMS721 Phase...

2013-10-30 08:31:24

SOUTH SAN FRANCISCO, Calif., Oct. 30, 2013 /PRNewswire/ -- KaloBios Pharmaceuticals, Inc. (Nasdaq: KBIO) today announced that the U.S. Food & Drug Administration (FDA) has granted the company orphan drug designation for KB001-A, an anti-PcrV monoclonal antibody (mAb) fragment, for the treatment of cystic fibrosis (CF) patients with Pseudomonas aeruginosa (Pa). (Logo: http://photos.prnewswire.com/prnh/20130225/MM66380LOGO) KaloBios is currently enrolling patients in a Phase 2...

2013-10-29 04:21:10

STOCKHOLM, Sweden, October 29, 2013 /PRNewswire/ -- Pharmalink AB, a specialty pharma company focused on orphan and niche products, today announces its product Busulipo(TM), a best-in-class conditioning agent for use in cancer patients prior to hematopoietic stem cell transplantation (HSCT; also known as bone marrow transplantation), has received orphan drug designation from the US Food and Drug Administration (FDA). The Company is currently preparing for registration trials...

2013-10-23 20:22:14

ReportsnReports.com adds Global RNAi Drug Pipeline Analysis 2013 market research report by PNS Pharma to its store. The research gives comprehensive insight on the various drugs being developed based upon RNAi technology DALLAS, Oct. 23, 2013 PRNewswire-iReach/ -- Global RNAi Drug Pipeline Analysis 2013 market research report covers all the ongoing drugs being developed in various development phases. Each drug profiles include detailed information like: Originator, Owner, Collaborator,...

2013-10-21 12:27:50

PRINCETON, N.J., Oct. 21, 2013 /PRNewswire/ -- Guerbet LLC, USA -- Guerbet, a pioneer in the field of contrast agents with more than 80 years of experience, announced that it has been granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) for Lipiodol (Ethiodized Oil) Injection for management of patients with known hepatocellular carcinoma (HCC). Hepatocellular carcinoma prevalence in US is estimated to...