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Last updated on April 24, 2014 at 1:21 EDT

Latest Orphan drug Stories

2012-08-27 23:02:22

QED Clinical Services, a global niche CRO operating in more than 40 countries, has announced that it will address the increasing demand of biotech and pharma companies to develop drugs in rare disease areas by launching OrphanReach, a division dedicated to this increasingly important research area. Milton Keynes, UK (PRWEB) August 26, 2012 The company's CEO, Thomas Ogorka, summarised QED's strategy moving forward: "We can clearly see a strong industry trend for orphan drug development...

2012-08-22 02:21:56

PHILADELPHIA, Aug. 22, 2012 /PRNewswire/ -- The Intellectual Property & Science business of Thomson Reuters, the world's leading provider of intelligent information for businesses and professionals, today released the results of its study on orphan drugs, developed to treat rare diseases, finding that they have the potential to generate as much lifetime revenue as drugs used for more common health conditions. The findings are featured in the Thomson Reuters paper, "The Economic Power of...

2012-07-23 06:26:37

NEW YORK and JERUSALEM, July 23, 2012 /PRNewswire/ -- Chiasma (pronounced key-azma), Inc., a privately held biopharma company, announced that it closed a $38.5 million equity financing co-led by new investor Abingworth and current investor MPM Capital that also included investments from existing shareholders 7 Med Health Ventures, ARCH Venture Partners, F3 Ventures and Fredric Price, Chiasma's Chairman & CEO. The new investment (the "D" round) is expected to provide funding for the...

2012-07-17 06:27:08

RESEARCH TRIANGLE PARK, N.C., July 17, 2012 /PRNewswire/ -- Grifols, a global healthcare company based in Barcelona, Spain, today announced plans to initiate a safety trial of a novel, inhaled formulation of alpha1-proteinase inhibitor [Human] later this year. In April, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for Grifols' inhaled alpha1 formulation as a treatment for cystic fibrosis, an inherited disease that can cause life-threatening lung infections....

2012-07-17 06:26:34

TORRANCE, Calif., July 17, 2012 /PRNewswire/ -- Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine technology company, and subsidiary of Emmaus Life Sciences, Inc., today announced that the European Commission (EC) has granted Orphan Medicinal Product designation for the company's investigational drug Levoglutamide (L-glutamine) for the treatment of sickle cell disease. The EC designation follows the recommendation of the European Medicines Agency's (EMA) Committee for...

2012-07-09 14:23:13

Approval Culminates Years of Advocacy, Education and Relationship Building Led by NORD WASHINGTON, July 9, 2012 /PRNewswire-USNewswire/ -- Signed by President Obama today, the U.S. Food and Drug Administration (FDA) Safety and Innovation Act includes the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983. As the long-standing and recognized policy leader and advocate for the rare disease community, the National Organization for...

2012-06-28 02:27:50

QUEBEC CITY, June 28, 2012 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced that its Japanese partner Yakult Honsha ("Yakult") (Tokyo: 2267) has initiated a Phase 1 trial in multiple myeloma, a form of blood cancer, with perifosine, the Company's oral Akt/PI3K inhibitor drug. Yakult, who are sponsoring and conducting this trial in Japan, reported that a first patient has been treated with perifosine. This is an...

2012-06-21 14:22:04

PHILADELPHIA, June 21, 2012 /PRNewswire/ -- There's a drug available to treat the symptoms of 10-year-old Brooke Foster's life-threatening, rare disease, but it isn't for sale in the United States. Brooke's mom, Kelli Foster, struggled for years to get her daughter's disease, mastocytosis, diagnosed. Now the New Jersey mom is fighting to have a drug that can treat the symptoms -- severe allergic reactions to medications, flavoring, dyes, preservatives, temperature changes, fragrances --...

2012-06-13 10:24:25

LITTLE FALLS, N.J., June 13, 2012 /PRNewswire/ -- Yesterday, on June 12, 2012, Regenicin, Inc. (OTC Bulletin Board: RGIN) a biotechnology company specializing in the development of and commercialization of regenerative cell therapies to restore the health of damaged tissues and organs, issued a press release that the Food and Drug Administration (FDA) had granted Orphan Status for the PermaDerm(®) product. While the Company believes it was accurate in representing the FDA's...

2012-06-13 02:27:42

QUÉBEC CITY, June 13, 2012 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced that Brian H. Kushner, MD, of the Memorial Sloan-Kettering Cancer Center in New York, will be making a poster presentation on Phase 1 results for perifosine in neuroblastoma, at the Advances in Neuroblastoma Research Conference which will be held June 18-21, 2012, at the Fairmount Royal York Hotel in Toronto....