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    Urea Cycle Disorders (UCD)

    Patients with UCD, due to genetic defects, are lack of the enzyme needed to decompose ammonia in the body, thus excessive ammonia cannot be eliminated and accumulates in the body, which lead to complications. In the acute stage, patients may experience confusion, nausea, vomiting and drowsiness. In the chronic phase, patients may experience confusion, dizziness, drowsiness, learning disabilities and mental retardation.

    Urea Cycle Disorders (UCD)
    Bile Acid Synthesis Defect

    Bile acid synthesis defect is a rare genetic disease caused by a defiency of enzymes in the synthesis of bile acid. The disease often occurs on newborns or infants and is less severe in adults. In the early stage, jaundice often occurs along with growth retardation and steatorrhea. As the disease progresses, cholestasis gradually worsens, even to the point of cirrhosis.

    Bile Acid Synthesis Defect
    Peyronie's disease

    Peyronie's disease occurs on men with specific symptons of bending along with pain of genitals when erection. In cases where conservative treatment has failed, removal of the hard lumps may be considered, but its efficacy is not satisfactory. Operative treatment may damage and destroy spongy body and result in erection disability or intensified bending of phallus after operation.

    Peyronie's disease
    Cushing's syndrome (CS)

    Cushing syndrome is a clinical syndrome caused by the chronic oversecreation of glucocorticoid by adrenal cortex due to multiple reasons. Direct symptons are shown clinically, among which moon-shaped face, central obesity, acne, purple striae, hypertension are the most typical ones. More than half of the patients with Cushing's syndrome suffer from major depression, atypical depression and other types of mental and psychological disorder.

    Cushing's syndrome (CS)
    Erythropoietic Protoporphyria (EPP )

    EPP is a photodermatosis caused by congenital mistake of heme biosynthesis pathway. Once exposed to visible light, patients would suffer from skin burning and internal tissue damage, which would lead to severe pain on skin and lesion. Therefore, patients are forced to stay indoors and are deprived of social interaction and normal life from birth.

    Erythropoietic Protoporphyria (EPP )
    Pipeline products
    Product
    Indications Regions Partners Type of Collaboration Pre-Clinical Phase I Phase II Phase III NDA Launch
    Ravicti
    UCD Greater China, S.Korea,SE Asia Greater China Immedica/HZNP License and Development
    Ravicti

    Indication: RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with ureacycle disorders (UCDs) including deficiencies of carbamoyl phosphate synthetase I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g.,essential amino acids, arginine, citrulline, protein-free calorie supplements).

    Partner: Immedica Pharma AB

    Diacerein
    EB Greater China Twi License and Development
    Diacerein

    Indication:

    Partner:

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