Stable parenteral DNJ compositions
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.
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.