Latest Orphan drug Stories
CHICAGO, Feb. 22, 2011 /PRNewswire/ -- Siren Interactive, a relationship marketing agency specializing in rare diseases, announces the publication of "Uncommon Challenges; Shared Journeys," a collection of 13 personal stories offering intimate views into the lives of families affected by rare disorders. The narratives reveal how parents -- mothers in particular -- play critical roles in obtaining a correct diagnosis and appropriate treatment for children with rare diseases. The stories also...
CLAREMONT, Calif., Jan. 31, 2011 /PRNewswire/ -- Keck Graduate Institute (KGI) will celebrate Rare Disease Day on Feb. 28 by hosting its second annual workshop with the U.S. Food and Drug Administration (FDA) for pharmaceutical and biotech companies and academics to learn how to write and file an Orphan Drug designation application. Hosted by KGI's Center for Rare Disease Therapies, the goal of the workshop is to simplify and demystify the application process so more companies and...
Agency experts to provide guidance on applying for orphan drug designation SILVER SPRING, Md., Jan. 27, 2011 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration has scheduled its fourth orphan drug designation workshop for academics, biotechnology companies, and those unfamiliar with the process for Feb. 28 - March 1, 2011, in Claremont, Calif. in collaboration with Keck Graduate Institute. (Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO) The workshop, co-sponsored...
NEW YORK and CARMIEL, Israel, Nov. 29, 2010 /PRNewswire/ -- Pfizer Inc. (NYSE: PFE) and Protalix BioTherapeutics, Inc. (NYSE-Amex: PLX, TASE: PLX) today announced the submission of a Marketing Authorization Application to the European Medicines Agency for taliglucerase alfa, a plant-cell expressed form of glucocerebrosidase (GCD)for the treatment of Gaucher disease. Taliglucerase alfa was granted Orphan Designation by the European Commission for the treatment of Gaucher disease on March...
Letter: Open letter to prime minister David Cameron and health secretary Andrew LansleyAn open letter from 20 consultants and a patient group published on bmj.com today, calls on the prime minister to take action over a legal loophole that allows drug companies to make easy profits by licensing existing treatments for rare (orphan) diseases.They argue that the current situation concerning orphan drugs is not in the best interests of patients or the NHS and that the cost to the NHS is likely...
SAN FRANCISCO, Nov. 2, 2010 /PRNewswire/ -- Deloitte released a new report, "Innovative Strategies for Oncology Drug Development," highlighting specific drug development approaches that have enabled biotech companies to produce a substantial number of first-in-class compounds and more than $1 billion in oncology drugs while transforming the medical outlook for many cancer patients. "Scientific innovation has brought measurable rewards such as reduced development times, higher success rates...
BLAINVILLE, QC, Oct. 26 /PRNewswire/ - A new player has entered into the Canadian orphan drug landscape:Â Medunik Canada, with a mission to bring currently unavailable orphan therapies for Canadian individuals with rare diseases. Headquartered in the northern area of Montreal, Medunik Canada is the sister company of Duchesnay Inc., a specialty pharmaceutical company dedicated mainly to the health of pregnant women and their unborn babies. Duchesnay and Medunik Canada are part of...
SINGAPORE, Oct. 18 /PRNewswire/ -- S*BIO Pte Ltd today announced that the European Commission (EC) has granted orphan drug designation to SB1518, its potent and orally active JAK2 inhibitor for the treatment of primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF) and post-essential thrombocythemia myelofibrosis (PET-MF). SB1518 has also received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of the same disorder. S*BIO...
Agency experts to provide guidance on applying for orphan drug designation SILVER SPRING, Md., Oct. 13 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration has scheduled the third in a series of workshops on orphan drug designation for academics, biotechnology companies, and those unfamiliar with the process for Nov. 4-5, 2010, in Lansdowne, Va. (Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO) (Logo: http://www.newscom.com/cgi-bin/prnh/20090824/FDALOGO) The...
STOCKHOLM, October 13, 2010 /PRNewswire/ -- Pharmalink AB, a privately held Swedish pharmaceutical company, has received orphan drug designation by the US Food and Drug Administration (FDA) for its product candidate Nefecon (PL-56) for patients with IgA nephropathy, also referred to as Berger's Disease. This program is presently in clinical development phase II. Johan Haggblad, Managing director of Pharmalink AB said: "Today's news marks a significant milestone for Pharmalink, and...
