Eisai Inc., of Woodcliff Lake, N.J., said the European Commission granted orphan drug designation to multityrosine kinase inhibitor lenvatinib for the treatment of follicular and papillary thyroid cancer

Hyperion Therapeutics Inc., of South San Francisco, said the FDA notified the firm that Ravicti (glycerol phenylbutyrate) qualified for orphan drug exclusivity, giving the product exclusivity on the market for seven years from the date of approval on Feb. 1

The FDA approved Cystaran in October 2012 and granted the product orphan drug status...On Tuesday, the FDA also approved Raptor Pharmaceutical Corp.'s orphan drug Procysbi (RP103, cysteamine bitartrate delayed-release) to treat the larger indication of nephropathic cystinosis. (See BioWorld Today, May 1, 2013

Xenon Pharmaceuticals Inc., of Burnaby, British Columbia, and Teva Pharmaceutical Industries Ltd., of Jerusalem, said the FDA has granted orphan drug designation to XEN402 to treat pain associated with erythromelalgia, a rare autosomal dominant condition characterized by burning pain in the feet and hands

portfolio company, received orphan drug status from the FDA for ApoCell, a treatment based on inducing immune tolerance for preventing graft-vs.-host disease

portfolio company, received orphan drug status from the FDA for ApoCell, a treatment based on inducing immune tolerance for preventing graft-vs.-host disease

Insmed Inc., of Monmouth Junction, N.J., said Arikace, its liposomal amikacin for inhalation, received orphan drug designation from the FDA for the treatment of infections caused by nontuberculus mycobacteria (NTM

Plasma-derived C1 esterase inhibitor is an orphan drug approved by the FDA to treat or prevent the symptoms of hereditary angioedema

Cangene Corp., of Winnipeg, Manitoba, said the FDA approved its Botulism Antitoxin (equine), heptavalent (A, B, C, D, E, F, G) (BAT) product for treating suspected or documented exposure to the botulinum neurotoxin A, B, C, D, E, F or G. Cangene's BAT received orphan drug designation and now has seven years of marketing exclusivity

Proteo Inc., of Irvine, Calif., said the FDA granted orphan drug designation to Elafin for the prevention of inflammatory complications of transthoracic esophagectomy

GenSpera Inc., of San Antonio, said the FDA granted orphan drug designation to G-202 for the treatment of hepatocellular carcinoma...ProMetic Life Sciences Inc., of Laval, Quebec, said the FDA granted orphan drug status for its plasma purified human plasminogen drug for use in the treatment of hypoplasminogenemia, or Type I plasminogen deficiency (T1PD

After nearly shuttering in 2009 following the failure of its initial program, in 2011 Adventrx acquired privately held SynthRx Inc., of Houston, and its rheologic and antithrombotic agent, poloxamer (ANX-188), to treat sickle cell crisis in a pediatric population, for which the compound has orphan drug designation

Pluristem Therapeutics Inc., of Haifa, Israel, said its PLacental eXpanded (PLX) cells received FDA orphan drug status in aplastic anemia...The FDA previously granted orphan drug designation to Pluristem's PLX cells in Buerger's disease

Pluristem Therapeutics Inc., of Haifa, Israel, said its PLacental eXpanded (PLX) cells received FDA orphan drug status in aplastic anemia...The FDA previously granted orphan drug designation to Pluristem's PLX cells in Buerger's disease

SOV Therapeutics Inc., of Morrisville, N.C., said the FDA granted orphan drug designation for the use of oral testosterone undecanoate, a prodrug of testosterone, in the treatment of constitutional delay of growth and puberty (CDGP) in adolescent boys, ages 14 to 17

At all doses tested, ALX-0061 was well tolerated and the safety profile compared favorably to data reported for other biological DMARDs...To date, almost 50 percent of planned patients have been enrolled in about 30 European centers...In both studies the JE vaccine was shown to be highly immunogenic in children/adolescents age 2 months to younger than 18 years with a safety profile comparable to pediatric vaccines licensed for other diseases

Osiris Therapeutics Inc., of Columbia, Md., reported that the European Medicines Agency granted orphan drug status to Prochymal (remestemcel-L) as a treatment of acute graft-vs.-host disease (GvHD

OSE Pharma SA, of Paris, said the FDA granted orphan drug status to its lead candidate, OSE 2101, to treat non-small-cell lung cancer (NSCLC) in patients expressing HLA-A2...Verastem Inc., of Cambridge, Mass., filed for orphan drug designation for VS-6063 in the U.S

Eisai Inc., of Woodcliff Lake, N.J., said the FDA granted orphan drug designation to its investigational drug lenvatinib (E7080) for follicular, medullary, anaplastic and metastatic or locally advanced papillary thyroid cancer