Latest Orphan drug Stories
NOVATO, Calif., Oct. 5, 2011 /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 UX001 for the treatment of hereditary inclusion body myopathy (HIBM). UX001 is an extended release formulation of sialic acid (SA-ER) intended as a substrate replacement therapy for HIBM, a severe, progressive,...
Orphan drug receives second approval for rare disease SILVER SPRING, Md., Sept. 23, 2011 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration today approved Soliris (eculizumab) to treat patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke. (Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO) Atypical HUS accounts for 5 to 10 percent...
Dr Lise Aagaard has been appointed Editor-in-Chief of the new open access journal Orphan Drugs: Research and Reviews, published by Dove Medical Press Auckland, New Zealand (PRWEB) September 21, 2011 Dove Medical Press has announced the appointment of Dr Lise Aagaard as the Editor-in-Chief of the new journal Orphan Drugs: Research and Reviews. This international, peer-reviewed, open access journal will publish original research, reports, reviews and commentaries on all areas of the design...
National Organization for Rare Disorders Calls for Policy on Products for Rare Diseases WASHINGTON, Sept. 7, 2011 /PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) has submitted a Citizen's Petition to the Food and Drug Administration (FDA) requesting that a documented policy be established regarding the review of potential treatments for people with rare diseases. (Logo: http://photos.prnewswire.com/prnh/20110719/DC37656LOGO-b) NORD's request is being made in...
SurvivingMesothelioma.com has good news this summer for some of the people involved in researching and developing drugs and treatments for diseases like mesothelioma. (PRWEB) August 26, 2011 SurvivingMesothelioma.com has good news this summer for some of the people involved in researching and developing drugs and treatments for diseases like mesothelioma. Mesothelioma, a rare cancer caused by inhaling asbestos, is classified as an ‘orphan disease’ because it affects fewer...
ANNAPOLIS, Md., Aug. 22, 2011 /PRNewswire/ -- The Plasma Protein Therapeutics Association (PPTA) believes the Internal Revenue Services' issuance of temporary and proposed regulations implementing the annual pharmaceutical fee undermines patients with rare diseases and fails to take into account concerns expressed by leading consumer organizations and Congress. The Agency's narrow definition of an "orphan drug" disproportionally affects the highly specialized segment of biological plasma...
CAMBRIDGE, Mass., Aug. 1, 2011 /PRNewswire/ -- Merrimack Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted MM-398 orphan drug status for the treatment of pancreatic cancer. MM-398 is a novel, stable nanotherapeutic encapsulation of the marketed chemotherapy drug irinotecan. MM-398 is partnered with PharmaEngine, Inc. for development and commercialization in Taiwan under the designation PEP02. (Logo:...
SILVER SPRING, Md., Aug. 1, 2011 /PRNewswire-USNewswire/ -- Science, public health, and regulatory highlights from the U.S. Food and Drug Administration. Information in this document is designed for credentialed journalists. Release dates and times for items are current as of Aug. 1, 2011. (Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO) FDA News & Notes does not contain any regulatory or enforcement actions due to legal limitations. Meetings, Workshops and Congressional...
H.R. 2672 and S. 1423 would exempt all exclusively orphan drugs from the annual tax on pharmaceuticals ANNAPOLIS, Md., July 29, 2011 /PRNewswire-USNewswire/ -- The Plasma Protein Therapeutics Association (PPTA) applauds the introduction of bipartisan legislation, the Preserving Access to Orphan Drugs Act (H.R. 2672) and (S. 1423), which will safeguard the development of drugs and therapies that treat patients with rare diseases by eliminating barriers to innovation. "The majority of...
SAN FRANCISCO, July 18, 2011 /PRNewswire/ -- Nektar Therapeutics (Nasdaq: NKTR) today announced that the European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on its application for orphan medicinal product status for the company's lead oncology candidate, NKTR-102, for the treatment of women with ovarian cancer. "There is a significant unmet need for additional treatments to address ovarian cancer," said Carlo DiFonzo, PhD, Vice President of...
