Latest Orphan drug Stories

NES-ZIONA, Israel, Feb. 13, 2013 /PRNewswire/ -- PROLOR Biotech, Inc. (NYSE MKT: PBTH) today announced that the European Commission and the European Medicines Agency (EMA) have granted orphan drug designation to hGH-CTP, PROLOR's longer-acting version of human growth hormone in development for the treatment of growth hormone deficiency. PROLOR will receive 10 years of marketing exclusivity in Europe for hGH-CTP, beginning at product launch. This is the first time a growth...

BURLINGTON, Mass., Feb. 7, 2013 /PRNewswire/ -- Decision Resources, one of the world's leading research and advisory firms for pharmaceutical and healthcare issues, finds that the expected supplemental approval of Millennium (Takeda)/Johnson & Johnson's Velcade for the treatment of chronic graft versus host disease (GVHD) will be a significant driver of growth in the GVHD market through 2021. In particular, Velcade's uptake in the first-line treatment setting will displace...

WASHINGTON, Jan. 28, 2013 /PRNewswire/ -- Today ten families of patients with a rare disease called cystinosis and three foundations supporting cystinosis research tell their stories to policymakers on Capitol Hill in an effort to educate lawmakers about rare diseases like cystinosis and how upcoming federal spending decisions could impact the availability of medicines for rare diseases. Cystinosis is a metabolic lysosomal storage disease that is diagnosed in infancy and often...

PALO ALTO, Calif., Jan. 11, 2013 /PRNewswire/ -- Telik, Inc. (Nasdaq: TELK) was notified today that its product candidate, ezatiostat HCL (Telintra), has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of myelodysplastic syndrome (MDS). Orphan designation grants potential US market exclusivity to a drug for the treatment of a specified condition for a period of seven years following FDA marketing approval. Additional potential...

VANCOUVER, British Columbia, Jan. 7, 2013 /PRNewswire/ -- Del Mar Pharma today announced that the European Committee for Orphan Medicinal Products (COMP) has recommended the designation of VAL-083 as an orphan medicinal product for the treatment of glioma. The COMP recommendation will be ratified by the European Commission in accordance with Article 5(8) of Regulation (EC) No. 141/2000. Glioma is a type of tumor that starts in the brain or spine and includes glioblastoma multiforme...

Rare Disease Patient Advocates Celebrate 30(th) Anniversary Today WASHINGTON, Jan. 4, 2013 /PRNewswire-USNewswire/ -- Thirty years ago today, President Ronald Reagan signed the Orphan Drug Act into law to encourage the development of treatments for the millions of Americans with rare diseases. (Logo: http://photos.prnewswire.com/prnh/20110719/DC37656LOGO-b) The new law represented a triumph of patient advocates working with government partners, the media, and other supporters to...

SUNNYVALE, Calif., Jan. 3, 2013 /PRNewswire/ -- Icon Bioscience, Inc., (IBI), a privately held biopharmaceutical company that specializes in the development and commercialization of novel ophthalmic pharmaceuticals, today announced that melphalan intraocular injection, the company's investigational product for the treatment of retinoblastoma, has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA). Orphan Drug Designation is generally granted to...

WASHINGTON, Dec. 25, 2012 /PRNewswire-USNewswire/ -- Jack Klugman touched the lives of millions of Americans through his support for what ultimately became the Orphan Drug Act. By portraying on his Quincy, M.E. television show the plight of people with rare diseases with no treatment, Mr. Klugman focused a spotlight on the need for incentives to encourage the development of therapies for small patient populations. He did this in partnership with patients and patient advocates who...

AMES, Iowa, Nov. 14, 2012 /PRNewswire/ -- NewLink Genetics Corporation (Nasdaq: NLNK) today announced that the European Commission (EC) has designated HyperAcute-Pancreas® Immunotherapy (algenpantucel-L) as an orphan medicinal product under Regulation (EC) No. 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products. NewLink filed the application for orphan designation in June 2012. A positive opinion was adopted by the Committee for...

EXTON, Pa., Nov. 12, 2012 /PRNewswire/ -- Morphotek(® )Inc., a subsidiary of Eisai Inc., announced today that the United States Food and Drug Administration (FDA) has granted orphan drug designation to its investigational cancer drug, amatuximab (MORAb-009) for the treatment of malignant pleural mesothelioma, the most common form of mesothelioma, which accounts for around 2,500 cases a year in the United States. This disease affects the pleura, which is the thin balloon shaped...