PHARMACEUTICAL COMPOSITIONS COMPRISING ETEPLIRSEN

1 . A pharmaceutical composition, comprising:

a) Eteplirsen;

b) sodium chloride;

c) potassium chloride;

d) potassium phosphate monobasic;

e) sodium phosphate dibasic; and

f) water,

wherein the concentration of Eteplirsen is about 50 mg/mL of the pharmaceutical composition.

2 . The pharmaceutical composition of claim 1 , comprising:

a) 40-60 mg of Eteplirsen;

b) 6.4-9.6 mg of sodium chloride;

c) 0.16-0.24 mg of potassium chloride;

d) 0.16-0.24 mg of potassium phosphate monobasic;

e) 0.91-1.37 mg of sodium phosphate dibasic; and

f) water,

wherein the concentration of Eteplirsen is about 50 mg/mL of the pharmaceutical composition.

3 . The pharmaceutical composition of claim 2 , comprising about 50 mg of Eteplirsen.

4 . The pharmaceutical composition of claim 1 , comprising:

a) about 50 mg of Eteplirsen;

b) about 8 mg of sodium chloride;

c) about 0.2 mg of potassium chloride;

d) about 0.2 mg of potassium phosphate monobasic;

e) about 1.14 mg of sodium phosphate dibasic; and

f) water,

wherein the concentration of Eteplirsen is about 50 mg/mL of the pharmaceutical composition.

5 . The pharmaceutical composition of claim 1 , comprising:

a) 80-120 mg of Eteplirsen;

b) 12.8-19.2 mg of sodium chloride;

c) 0.32-0.48 mg of potassium chloride;

d) 0.32-0.48 mg of potassium phosphate monobasic;

e) 1.02-1.54 mg of sodium phosphate dibasic; and

f) water,

wherein the concentration of Eteplirsen is about 50 mg/mL of the pharmaceutical composition.

6 . The pharmaceutical composition of claim 5 , comprising about 100 mg of Eteplirsen.

7 . The pharmaceutical composition of claim 5 , comprising:

a) about 100 mg of Eteplirsen;

b) about 16 mg of sodium chloride;

c) about 0.4 mg of potassium chloride;

d) about 0.4 mg of potassium phosphate monobasic;

e) about 2.28 mg of sodium phosphate dibasic; and

f) water,

wherein the concentration of Eteplirsen is about 50 mg/mL of the pharmaceutical composition.

8 . The pharmaceutical composition of claim 1 , comprising:

a) 400-600 mg of Eteplirsen;

b) 64-96 mg of sodium chloride;

c) 1.6-2.4 mg of potassium chloride;

d) 1.6-2.4 mg of potassium phosphate monobasic;

e) 9.0-14.0 mg of sodium phosphate dibasic; and

f) water,

wherein the concentration of Eteplirsen is about 50 mg/mL of the pharmaceutical composition.

9 . The pharmaceutical composition of claim 8 , comprising about 500 mg of Eteplirsen.

10 . The pharmaceutical composition of claim 8 , comprising:

a) about 500 mg of Eteplirsen;

b) about 80 mg of sodium chloride;

c) about 2 mg of potassium chloride;

d) about 2 mg of potassium phosphate monobasic;

e) about 11.4 mg of sodium phosphate dibasic; and

f) water,

wherein the concentration of Eteplirsen is about 50 mg/mL of the pharmaceutical composition.

11 . The pharmaceutical composition of claim 1 , comprising:

a) about 5 w/v % Eteplirsen;

b) about 0.8 w/v % sodium chloride;

c) about 0.02 w/v % potassium chloride;

d) about 0.02 w/v % potassium phosphate monobasic;

e) about 0.114 w/v % sodium phosphate dibasic; and

f) water.

12 . The pharmaceutical composition of claim 1 , comprising:

a) about 50 mg/mL Eteplirsen;

b) about 8 mg/mL sodium chloride;

c) about 0.2 mg/mL potassium chloride;

d) about 0.2 mg/mL potassium phosphate monobasic;

e) about 1.14 mg/mL sodium phosphate dibasic; and

f) water.

13 . The pharmaceutical composition according to any one of claims 1 - 12 , wherein the pH of the pharmaceutical composition is about 7.5, and the osmolality of the pharmaceutical composition ranges from about 260 mOsm to about 320 mOsm.

14 . A method for treating Duchenne muscular dystrophy (DMD) in a subject in need thereof wherein the subject has a mutation of the dystrophin gene that is amenable to exon 51 skipping, comprising administering to the subject a pharmaceutical composition of any one of claims 1 - 13 .

15 . Use of the pharmaceutical composition of any one of claims 1 - 13 for the manufacture of a medicament for the treatment Duchenne muscular dystrophy (DMD) in a subject in need thereof, wherein the subject has a mutation of the dystrophin gene that is amenable to exon 51 skipping.