Compositions for an orally active 1,2,4-oxadiazole for the treatment of disease

1. A method for treating a patient having aniridia associated with a lack of expression of a gene resulting from a premature stop codon or a nonsense mutation, comprising administering to the patient an oral pharmaceutical composition comprising 25.0% by weight of 3-[5-(2-fluoro-phenyl)[1,2,4]oxadiazol-3-yl]-benzoic acid or a pharmaceutically acceptable salt thereof; 25.7% by weight of refined polydextrose; 25.0% by weight of mannitol; 12.8% by weight of a surfactant selected from either or both polyethylene glycol 3350 and 3.7% by weight of poloxamer 407 powder; 5.0% by weight of crospovidone; and less than 2% by weight of one or more excipients selected from hydroxyethyl cellulose, vanilla flavor, non-bovine magnesium stearate or colloidal silica, wherein the method comprises administration of three doses of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid or a pharmaceutically acceptable salt thereof in a 24 hour period according to the formula: lx, lx, 2X, and wherein said administration maintains a plasma concentration of 3-[5-(2-fluoro-phenyl)[1,2,4]oxadiazol-3-yl]-benzoic acid or a pharmaceutically acceptable salt thereof in a range of from about 2μg/mL to about 20 μg/mL in said patient for the 24 hour period.

2. The method of claim 1 , wherein said oral pharmaceutical composition comprises from 35 mg to 1400 mg of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-y1]-benzoic acid or a pharmaceutically acceptable salt thereof in a unit dosage formulation.

3. The method of claim 2 , wherein the amount of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid or a pharmaceutically acceptable salt thereof in the unit dosage formulation is selected from 35 mg, 50 mg, 70 mg, 100 mg, 125 mg, 140 mg, 175 mg, 200 mg, 250 mg, 280 mg, 350 mg, 500 mg, 560 mg, 700 mg, 750 mg, 1000 mg, or 1400 mg.

4. The method of claim 1 , wherein the oral pharmaceutical composition is a unit dosage formulation packaged in plastic bottles.

5. The use of claim 1 , wherein the oral pharmaceutical composition is a powder or granules packaged in a sachet.

6. The method of claim 1 , wherein the oral pharmaceutical composition is reconstituted as a suspension to achieve a concentration selected from 25 mg/mL in the total volume or 50 mg/mL in the total volume.

7. The method of claim 1 , wherein the oral pharmaceutical composition is reconstituted using water, milk, a carbonated beverage, juice, apple sauce, baby food or baby formula.

8. The method of claim 1 , wherein the three doses are administered orally to a human.

9. The method of claim 1 , wherein the first dose is administered in the morning, the second dose is administered at midday and the third dose is administered in the evening of the 24 hour period.

10. The method of claim 1 , wherein the second dose is administered about 6 hours after the first dose is administered, wherein the third dose is administered about 6 hours after the second dose is administered and wherein the first dose is administered about 12 hours after the third dose was administered in a preceding 24 hour period, and wherein the first dose, the second dose, or the third dose are administered about 30 minutes after a meal.

11. The method of claim 1 , wherein the three daily doses are administered in a continuous therapy comprising a plurality of 24 hour periods.

12. The method of claim 1 , wherein X is about 8 mg/kg to about 12 mg/kg.

13. The method of claim 12 , wherein X is about 10 mg/kg.