Claims for Patent: 11,229,661

Summary for Patent: 11,229,661

Title:	Bile acid recycling inhibitors for treatment of pediatric cholestatic liver diseases
Abstract:	Provided herein are methods of treating or ameliorating a pediatric cholestatic liver disease by non-systemically administering to an individual in need thereof a therapeutically effective amount of a pediatric formulation comprising an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI) or a pharmaceutically acceptable salt thereof. Also provided are methods for treating or ameliorating a pediatric liver disease, decreasing the levels of serum bile acids or hepatic bile acids, treating or ameliorating pruritis, reducing liver enzymes, or reducing bilirubin comprising non-systemically administering to an individual in need thereof a therapeutically effective amount of a pediatric formulation comprising an ASBTI or a pharmaceutically acceptable salt thereof.
Inventor(s):	Gedulin Bronislava, Grey Michael, "ODonnell Niall"
Assignee:	SHIRE HUMAN GENETIC THERAPIES, INC.
Application Number:	US16679864
Patent Claims:	2. The method of claim 1 , wherein the ASBTI is effective for decreasing at least 20% of serum and/or hepatic bile acid levels in the pediatric subject as compared to bile acid levels prior to administration of the ASBTI.3. The method of claim 1 , wherein the pediatric disorder is characterized by having a BSEP deficiency.4. The method of claim 1 , wherein the pediatric subject has a mutation in the ABCB11 gene.5. The method of claim 1 , wherein the pediatric disorder is PFIC2.6. The method of claim 1 , wherein the pediatric patient is between 6 months and 12 years old.7. The method of claim 1 , wherein less than 10% of the ASBTI is systemically absorbed.8. The method of claim 1 , wherein the ASBTI is effective for decreasing at least 30% of serum and/or hepatic bile acid levels in the pediatric subject as compared to bile acid levels prior to administration of the ASBTI.9. The method of claim 1 , wherein the ASBTI is effective for decreasing at least 40% of serum and/or hepatic bile acid levels in the pediatric subject as compared to bile acid levels prior to administration of the ASBTI.10. The method of claim 1 , wherein the ASBTI is effective for decreasing at least 50% of serum and/or hepatic bile acid levels in the pediatric subject as compared. to bile acid levels prior to administration of the ASBTI.11. The method of comprising administering to the pediatric subject a pharmaceutical composition comprising the ASBTI claim 1 , or the pharmaceutically acceptable salt thereof.12. The method of claim 11 , wherein the composition is a pediatric dosage form.13. The method of claim 12 , wherein the pediatric dosage form is selected from a solution claim 12 , syrup claim 12 , suspension claim 12 , elixir claim 12 , powder for reconstitution as suspension or solution claim 12 , dispersible/effervescent tablet claim 12 , chewable tablet claim 12 , gummy candy claim 12 , lollipop claim 12 , freezer pops claim 12 , troches claim 12 , oral thin strips claim 12 , orally disintegrating tablet claim 12 , sachet claim 12 , soft gelatin capsule claim 12 , and sprinkle oral powder or granules.14. The method of claim 11 , wherein he composition comprises between about 1 mg/kg to about 50 mg/kg of the ASBTI.15. The method of claim 11 , wherein the composition comprises between about 0.001 mg/kg to about 10 mg/kg of the ASBTI.16. The method of claim 12 , wherein the pediatric dosage form comprises between 0.1 to 40 mg of the ASBTI.17. The method of claim 11 , wherein the composition further comprises a bile acid sequestrant or binder.