Abstract: Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.
Abstract: The present invention features pharmaceutical compositions including sepiapterin, or a pharmaceutically acceptable salt and/or co-crystal thereof, and methods for the treatment of tetrahydrobiopterin-related disorders (e.g., tetrahydrobiopterin deficiency or phenylketonuria) with such compositions.
Abstract: The present invention features compositions including sepiapterin, or a pharmaceutically acceptable salt thereof, and methods for the treatment of BH4-related disorders. In some embodiments, these compositions and methods result in an increase in plasma, CSF, and/or brain exposure of sepiapterin.
Abstract: Disclosed is a means for improving the symptoms of a cerebral dysfunction. The present inventors made a new discovery that the activity of brain aromatic monoamines increases when sepiapterin is administered peripherally. Disclosed, therefore, is a medicinal agent, which contains at least sepiapterin or a salt thereof, for preventing or improving cerebral dysfunction. Also disclosed is a beverage/food, which contains at least sepiapterin or a salt thereof, for preventing or improving cerebral dysfunction. Unlike tetrahydrobiopterin and the like, sepiapterin can control reductions in the brain neuron levels of brain aromatic monoamines (serotonin, dopamine, noradrenaline, and the like) and increase the activity thereof even when administered peripherally.
Abstract: Disclosed is a crystalline form of sepiapterin, a method of preparing the crystalline form, pharmaceutical compositions containing the crystalline form, and a method for treating patients with a disease associated with low intracellular BH4 levels or with dysfunction of various BH4 dependent metabolic pathways, which involves administering to the patient an effective amount of the crystalline form.
Type:
Grant
Filed:
November 28, 2017
Date of Patent:
September 28, 2021
Assignee:
PTC Therapeutics MP, Inc.
Inventors:
Hiroshi Yoshino, Taichi Komoda, Yuichi Shiro, Shunichi Murata, Takayoshi Matsumoto, Kaito Kishimoto, Daniel E. Levy
Abstract: Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.