The compound has been granted orphan drug designation in the U.S

Arno Therapeutics Inc., of Flemington, N.J., said the European Medicines Agency granted orphan drug status to AR-42 in neurofibromatosis Type 2, a rare genetic disorder characterized by the growth of noncancerous tumors in the brain and spinal cord, juvenile cataracts and neurofibromas of the skin

Hematech will pay $10 million ($1 million up front and $9 million in staged payments) to fund the unnamed orphan drug's development program up to regulatory approval

Sunesis Pharmaceuticals Inc., of South San Francisco, said the European Commission granted orphan drug designation to vosaroxin, the company's lead development candidate, in acute myeloid leukemia (AML...Vosaroxin previously received orphan drug and fast-track designations from the FDA

Medgenics Inc., of Misgav, Israel, filed for FDA orphan drug designation for Infradure in hepatitis D. Infradure seeks to replace interferon-alpha injections for hepatitis D by using Medgenics' tissue-based Biopump platform, which uses the patient's own tissue to continuously produce and deliver therapeutic proteins...The commission has granted orphan drug designation to Jakavi, which Novartis licensed from Incyte Corp., of Wilmington, Del., for development and commercialization outside the U.S

Medgenics Inc., of Misgav, Israel, filed for FDA orphan drug designation for Infradure in hepatitis D. Infradure seeks to replace interferon-alpha injections for hepatitis D by using Medgenics' tissue-based Biopump platform, which uses the patient's own tissue to continuously produce and deliver therapeutic proteins

The commission has granted orphan drug designation to Jakavi, which Novartis licensed from Incyte Corp., of Wilmington, Del., for development and commercialization outside the U.S. Incyte retains rights to the drug in the U.S., where it was the first JAK inhibitor to be approved

NeuroSearch A/S, of Ballerup, Denmark, said it is seeking financing to conduct the Prime-HD study of Huntexil (pridopidine), an orphan drug in Phase III development for the treatment of Huntington's disease

NeuroSearch A/S, of Ballerup, Denmark, said it is seeking financing to conduct the Prime-HD study of Huntexil (pridopidine), an orphan drug in Phase III development for the treatment of Huntington's disease

Threshold Pharmaceuticals Inc., of South San Francisco, received orphan drug designation in the U.S...The drug, which received orphan drug designation in Europe last month, is in a pivotal Phase III trial that is comparing it in combination with doxorubicin against doxorubicin alone

Isis Pharmaceuticals Inc., of Carlsbad, Calif., said partner Cambridge, Mass.-based Genzyme, a unit of Sanofi SA, submitted a new drug application (NDA) for Kynamro (mipomersen sodium) in the orphan drug indication homozygous familial hypercholesterolemia (HoFH

Ultragenyx Pharmaceutical Inc., of Novato, Calif., received orphan drug designation from the European Medicines Agency for sialic acid to treat hereditary inclusion body myopathy (HIBM) and recombinant human beta-glucuronidase for mucopolysaccharidosis Type 7 (MPS7...Both drugs also have orphan drug status from the FDA

Lexicon Pharmaceuticals Inc., of The Woodlands, Texas, received orphan drug designation from the FDA for its candidate for carcinoid syndrome, telotristat etiprate (LX1032

The FDA has granted orphan drug designation to the compound, and the company plans to begin a Phase II study in the second half of 2012

QLT Inc., of Vancouver, British Columbia, said its commercial product Visudyne (verteprofin) was granted orphan drug designation by the FDA for the potential treatment of chronic or recurrent central serous chorioretinopathy (CSC

Threshold Pharmaceuticals Inc., of South San Francisco, said the European Commission granted orphan drug designation to TH-302, a hypoxia-targeted drug, in soft-tissue sarcoma

Lomitapide is a small-molecule microsomal triglyceride transfer protein inhibitor that has orphan drug status for HoFH in the U.S. Shares of Aegerion (NASDAQ:AEGR) gained $1.44, or 10.4 percent, to close Thursday at $15.30...Promedior Inc., of Malvern, Penn., said the FDA granted orphan drug status to PRM-151 , a recombinant form of human Pentraxin-2, in idiopathic pulmonary fibrosis

Cytokinetics Inc., of South San Francisco, received orphan drug designation from the European Medicines Agency for CK-2017357, a fast skeletal muscle troponin activator in Phase II trials for amyotrophic lateral sclerosis...CK-2017357 is the lead drug candidate in Cytokinetics' skeletal sarcomere activator program and previously received orphan drug designation in the U.S

Can-Fite BioPharma Ltd., of Petah-Tikva, Israel, said the FDA granted orphan drug status to CF102, its candidate in hepatocellular carcinoma (primary liver cancer