Therapeutic Agent for Bh4-Responsive Hyperphenylalaninemia
    2.
    发明申请
    Therapeutic Agent for Bh4-Responsive Hyperphenylalaninemia 审中-公开
    Bh4反应性高苯丙氨酸血症的治疗剂

    公开(公告)号:US20080207624A1

    公开(公告)日:2008-08-28

    申请号:US11579928

    申请日:2005-05-11

    IPC分类号: A61K31/519

    摘要: A pharmaceutical preparation containing sapropterin hydrochloride can be used to administer sapropterin hydrochloride, an effective therapeutic agent for the treatment of tetrahydrobiopterin-responsive hyperphenylalaninemia, to patients of a wide range of ages, ranging from infants to adults, in a single preparation. One specific example is a pharmaceutical preparation for the treatment of BH4-responsive hyperphenylalaninemia provided in the form of granule, fine granule, or dry syrup, comprising sapropterin hydrochloride as an active ingredient; a flavoring agent; a coloring agent which is stable to acid and oxidation; and ascorbic acid or L-cysteine hydrochloride as a stabilizer, wherein the preparation has a moisture content (weight loss on drying) of 0.9% or less. Also provided is the pharmaceutical preparation in which the amount of sapropterin hydrochloride is from 2.5 to 20 wt %.

    摘要翻译: 含有盐酸沙曲肽的药物制剂可以用于在单一制剂中给予盐酸沙曲肽盐酸盐,其是用于治疗四氢生物喋呤反应型高苯丙氨酸血症的有效治疗剂,其范围广泛,范围从婴儿到成人。 一个具体实例是用于治疗以颗粒,细颗粒或干糖浆形式提供的BH4应答性高苯丙氨酸血症的药物制剂,其包含盐酸沙曲肽作为活性成分; 调味剂; 对酸和氧化稳定的着色剂; 和抗坏血酸或L-半胱氨酸盐酸盐作为稳定剂,其中所述制剂的水分含量(干燥时的重量损失)为0.9%以下。 还提供了其中盐酸沙普硝酸盐的量为2.5〜20重量%的药物制剂。