Sobi Canada Inc., a subsidiary of Swedish Orphan Biovitrum AB (publ) (Sobi), announced that Health Canada has approved Orfadin (nitisinone) capsules for the treatment of hereditary tyrosinaemia type-1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. HT-1 is a rare genetic disease that affects infants and children. It is progressive and may result in liver, brain and kidney complications and can be fatal if untreated.
Senhwa Biosciences, Inc. announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to CX-4945 for the treatment of cholangiocarcinoma.
Alexion Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to ALXN1210, a highly innovative, longer-acting anti-C5 antibody that inhibits terminal complement, which is being evaluated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a debilitating, ultra-rare, life-threatening blood disorder in which uncontrolled activation of complement, a component of the immune system, results in hemolysis (destruction of a patient’s red blood cells).
Adamas Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for ADS-5102 (amantadine hydrochloride) extended-release capsules, for the treatment of levodopa-induced dyskinesia (LID) in patients with Parkinson's disease. The ADS-5102 application has been given a Prescription Drug User Fee Act (PDUFA) target action date of August 24, 2017.
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