Claims for Patent: 12,257,252

Summary for Patent: 12,257,252

Title:	Compositions and methods for increasing tetrahydrobiopterin plasma exposure
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 exposure of BH4.
Inventor(s):	Neil Smith, Jonathan Reis
Assignee:	PTC Therapeutics MP Inc
Application Number:	US17/059,632
Patent Claims:	1. A method of therapeutic treatment of a BH4-related disorder in a subject in need thereof, the method comprising administering to the subject an effective amount of sepiapterin, or a pharmaceutically acceptable salt thereof, with food, wherein the BH4-related disorder is a BH4 deficiency, high plasma phenylalanine, or phenylketonuria. 2. The method of claim 1, wherein the effective amount is an amount sufficient to produce a concentration of at least 50 ng/ml in the plasma of the subject within 10 hours of administration. 3. The method of claim 2, wherein the effective amount comprises a dose that is at least 20% lower than the dose sufficient to produce a maximum BH4 plasma concentration (Cmax) of at least 50 ng/mL in the plasma of the subject within 10 hours of administration of sepiapterin, or a pharmaceutically acceptable salt thereof, without food. 4. The method of claim 1, wherein the effective amount is 2.5 mg/kg to 100 mg/kg per dose. 5. The method of claim 1, wherein the administration to the subject occurs less than 30 minutes prior to consuming food. 6. The method of claim 1, wherein the administration to the subject is substantially at the same time as food. 7. The method of claim 1, wherein the administration is immediately after the consumption of food up to 2 hours after the consumption. 8. The method of claim 1, wherein the effective amount results in an increase in the maximum plasma concentration (Cmax) of BH4 compared to administration without food. 9. The method of claim 1, wherein the effective amount results in an increase in the area under the concentration time curve from time zero to last concentration (AUC0-last) of BH4 compared to administration without food. 10. The method of claim 1, wherein the BH4-related disorder is a BH4 deficiency. 11. The method of claim 10, wherein the BH4 deficiency is primary BH4 deficiency. 12. The method of claim 1, wherein the BH4-related disorder is phenylketonuria. 13. The method of claim 1, wherein the BH4-related disorder is high plasma phenylalanine.