# US 10,117,830 B2 · Stable parenteral DNJ compositions

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- Number: US 10,117,830 B2
- Document ID: US10117830B2
- Status: Granted
- Kind: B2
- Application number: 15/822,655
- Filed: 2017-11-27
- Published: 2018-03-22
- Granted: 2018-11-06
- Earliest claimed: 2013-01-09
- Assignees: Amicus Therapeutics, Inc.
- Applicants: Amicus Therapeutics, Inc.
- Inventors: Hing Char; Sergey Tesler; Jiping Yang; Enrique Dilone
- Web page: https://patentgrep.com/patent/US10117830B2

## CPC

- A61K 9/0019: {Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00)}
- A61K 31/445: Non condensed piperidines, e.g. piperocaine
- A61K 31/45: having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
- A61K 47/02: Inorganic compounds
- A61K 47/12: Carboxylic acids; Salts or anhydrides thereof
- A61K 47/183: {Amino acids, e.g. glycine, EDTA or aspartame}
- A61P 9/00: Drugs for disorders of the cardiovascular system
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 25/00: Drugs for disorders of the nervous system
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## 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.

## Claims

### Claim 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.

### Claim 2 (depends on 1)

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

### Claim 3 (depends on 1)

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

### Claim 4 (depends on 1)

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

### Claim 5 (depends on 1)

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

### Claim 6 (depends on 1)

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

### Claim 7 (depends on 1)

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.

### Claim 8 (depends on 1)

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.

### Claim 9 (depends on 1)

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

### Claim 10 (depends on 1)

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

### Claim 11 (depends on 1)

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

### Claim 12 (depends on 1)

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

### Claim 13 (depends on 12)

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

### Claim 14 (depends on 13)

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.

### Claim 15 (depends on 1)

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.

### Claim 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.

### Claim 17 (depends on 16)

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

### Claim 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.

### Claim 19 (depends on 18)

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

### Claim 20 (depends on 18)

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