Stable parenteral DNJ compositions

Patent metadata

Field	Value
Publication number	US-10117830-B2
Application number	US-201715822655-A
Country	US
Kind code	B2
Filing date	Nov 27, 2017
Priority date	Jan 9, 2013
Publication date	Nov 6, 2018
Grant date	Nov 6, 2018

Abstract

A stable pharmaceutical composition that includes an active agent selected from 1-deoxynojirimycin, a pharmaceutically acceptable salt thereof, or a derivative thereof, and a buffer, wherein the stable pharmaceutical composition is capable of being parenterally administered to a human without deleterious health effects. Pompe disease is an example of a lysosomal storage disorder. Pompe disease is caused by a deficiency in the enzyme acid alpha-glucosidase (GAA). GAA metabolizes glycogen, a storage form of sugar used for energy, into glucose.

First claim

The invention claimed is:

1. A pharmaceutical composition comprising:
a) an active agent selected from 1-deoxynojirimycin, a pharmaceutically acceptable salt thereof, or a derivative thereof, and
b) a buffer,

wherein the active agent is present at a concentration of from about 1 mg/mL to about 100 mg/mL.

2. The pharmaceutical composition of claim 1 , wherein the active agent comprises 1-deoxynojirimycin.

3. The pharmaceutical composition of claim 1 wherein the active agent comprises 1-deoxynojirimycin hydrochloride.

4. The pharmaceutical composition of claim 1 , wherein the active agent comprises N-butyl-deoxynojirimycin, or a pharmaceutically acceptable salt thereof.

5. The pharmaceutical composition of claim 1 , wherein the pharmaceutical composition is formulated for intravenous administration.

6. The pharmaceutical composition of claim 1 , wherein the pharmaceutical composition is at a pH of from about 4 to about 6.

7. The pharmaceutical composition of claim 1 , wherein the active agent is present at a concentration of from about 1 mg/mL to about 60 mg/mL.

8. The pharmaceutical composition of claim 1 , wherein the active agent is present at a concentration of from about 25 mg/mL to about 30 mg/mL.

9. The pharmaceutical composition of claim 1 , wherein the buffer is present at a concentration of about 20 mM to about 75 mM.

10. The pharmaceutical composition of claim 1 , wherein the buffer is present at a concentration of about 40 mM to about 50 mM.

11. The pharmaceutical composition of claim 1 , wherein the buffer comprises a citrate buffer.

12. The pharmaceutical composition of claim 1 , further comprising a chelating agent.

13. The pharmaceutical composition of claim 12 , wherein the chelating agent comprises EDTA.

14. The pharmaceutical composition of claim 13 , wherein the EDTA is present at a concentration of about 0.005% to about 0.25% weight by volume.

15. The pharmaceutical composition of claim 1 , wherein the pharmaceutical composition is shelf-stable for at least about one year under an atmosphere selected from nitrogen, air or a combination thereof, and a temperature of from about 2° C. to about 42° C.

16. A pharmaceutical composition comprising
a) an active agent comprising N -butyl-deoxynojirimycin, or a pharmaceutically acceptable salt thereof , and
b) a buffer,

wherein the active agent is present at a concentration of from about 1 mg/mL to about 100 mg/mL and the buffer is present at a concentration of about 20 mM to about 75 mM.

17. The pharmaceutical composition of claim 16 , wherein the buffer comprises a citrate buffer.

18. A method for increasing the stability of a formulation comprising an active agent selected from 1-deoxynojirimycin, a pharmaceutically acceptable salt thereof, or a derivative thereof, the method comprising introducing a buffer into the formulation, wherein the active agent is present at a concentration of from about 1 mg/ml to about 100 mg/mL.

19. The method of claim 18 , wherein the active agent comprises N-butyl-deoxynojirimycin, or a pharmaceutically acceptable salt thereof.

20. The method of claim 18 , wherein the buffer comprises a citrate buffer.

Assignees

• Amicus Therapeutics Inc

Inventors

• Char Hing
• Tesler Sergey
• Yang Jiping
• Dilone Enrique

Classifications

• A61P43/00

  Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title

• A61P9/00

  Drugs for disorders of the cardiovascular system · CPC title

• A61P21/00

  Drugs for disorders of the muscular or neuromuscular system · CPC title

• A61P25/00

  Drugs for disorders of the nervous system · CPC title

• A61K47/12

  Carboxylic acids; Salts or anhydrides thereof · CPC title