Granted Patent
B2
US 12,329,757 · App. 18/129,177 · Granted Jun 17, 2025
Methods for increasing sepiapterin plasma exposure
Assignee:
PTC Therapeutics MP, Inc.
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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.
Claims (6)
1. A method of treating 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, without food, wherein the BH4-related disorder is phenylketonuria.
2. The method of claim 1 , wherein the effective amount is an amount sufficient to produce a concentration of at least 0.5 ng/mL in the plasma of the subject within 1 hour 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 plasma concentration (Cmax) of at least 0.5 ng/mL in the subject within 1 hour of administration of sepiapterin, or a pharmaceutically acceptable salt thereof, with 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 more than 30 minutes before or more than four hours after consuming food.
6. The method of claim 1 , wherein the effective amount results in an increase in the maximum plasma, CSF, and/or brain concentration (Cmax) of sepiapterin, or a pharmaceutically acceptable salt thereof, compared to administration with food.
Assignments (3)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded May 15, 2024
From: SMITH, NEIL; REIS, JONATHAN
To: CENSA PHARMACEUTICALS INC.
Reel/Frame 067422/0252 →
CHANGE OF NAME
Recorded May 15, 2024
From: CENSA PHARMACEUTICALS INC.
To: PTC THERAPEUTICS MP, INC.
Reel/Frame 067427/0748 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Sep 20, 2023
From: SMITH, NEIL; REIS, JONATHAN
To: CENSA PHARMACEUTICALS INC.
Reel/Frame 064963/0448 →
Priority Claims (1)
GC 2019/37663
· May 28, 2019
· regional
Continuity (5)
Continuation
17059719
Provisional Application
62822376
· Mar 22, 2019
Provisional Application
62771398
· Nov 26, 2018
Provisional Application
62677943
· May 30, 2018
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Cited By (1)