PHARMACEUTICAL COMPOSITIONS COMPRISING SEPIAPTERIN AND USES THEREOF
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.
1 . A pharmaceutical composition comprising sepiapterin, or a pharmaceutically acceptable salt thereof, wherein the pharmaceutical composition is substantially free of antioxidant.
2 - 4 . (canceled)
5 . The pharmaceutical composition of claim 1 , wherein the sepiapterin, or a pharmaceutically acceptable salt thereof, is in crystalline form.
6 . The pharmaceutical composition of claim 5 , wherein the crystalline form of is a crystalline form of sepiapterin free base having peaks at diffraction angle 2θ (°) of about 9.7°, about 10.2°, and about 11.3° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry.
7 . The pharmaceutical composition of claim 1 , wherein the pharmaceutical composition comprises less than 1.3% of lactoylpterin by weight of the combined amount of sepiapterin and lactoylpterin in the composition.
8 . The pharmaceutical composition of claim 1 , wherein the pharmaceutical composition is stable at room temperature for at least 6 months.
9 . A method of treating phenylketonuria in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition of claim 1 .
10 . A method of decreasing phenylalanine levels in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition of claim 1 .
11 . A method of increasing the activity of phenylalanine hydroxylase in a subject, the method comprising administering to the subject an effective amount of a pharmaceutical composition of claim 1 .
12 . A method of increasing serotonin levels in a subject, the method comprising administering to the subject an effective amount of a pharmaceutical composition of claim 1 .
13 . A method of increasing dopamine levels in a subject, the method comprising administering to the subject an effective amount of a pharmaceutical composition of claim 1 .
14 . A method of treating a tetrahydrobiopterin deficiency in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition of claim 1 .
15 . The method of claim 14 , wherein the tetrahydrobiopterin deficiency is primary tetrahydrobiopterin deficiency.
16 - 18 . (canceled)
19 . The pharmaceutical composition of claim 1 , wherein the pharmaceutical composition is a solid pharmaceutical composition.
20 . The pharmaceutical composition of claim 19 , formulated as a powder.