C1-INH compositions and methods for the prevention and treatment of disorders associated with C1 esterase inhibitor deficiency
Compositions and methods for the treatment and/or prevention of disorders associated with C1 esterase inhibitor deficiency are disclosed.
1. A method for treating hereditary angioedema (HAE), said method comprising subcutaneously administering to a subject in need thereof a composition comprising a C1 esterase inhibitor, a buffer selected from citrate or phosphate, and having a pH ranging from 6.5-8.0, wherein the C1 esterase inhibitor is administered at a concentration of at least about 400 U/mL and a dose of at least about 1000 U, and wherein the administration of the composition comprising the C1 esterase inhibitor increases the level of C1 esterase inhibitor in the blood of the subject to at least about 0.4 U/mL, and wherein the C1 esterase inhibitor comprises an amino acid sequence at least 95% identical to residues 23 to 500 of SEQ ID NO:1.
2. The method of claim 1 , wherein the C1 esterase inhibitor is present in the composition in a concentration of at least about 500 U/mL in the composition.
3. The method of claim 1 , wherein the composition is administered daily, every other day, or every three days.
4. The method of claim 1 , wherein the composition is administered one, two, or three times a week.
5. The method of claim 1 , wherein the increased blood levels of C1 esterase inhibitor are maintained at least 50% of the time between administrations.
6. The method of claim 1 , wherein the HAE is Type I HAE or Type II HAE.
7. The method of claim 1 , wherein the administration of the composition comprising a C1 esterase inhibitor results in HAE prophylactic treatment.
8. The method of claim 1 , wherein the administration of the composition comprising a C1 esterase inhibitor results in treatment of an HAE attack.
9. The method of claim 1 , wherein the administration of the composition results in at least a reduction in the severity and/or number of HAE attacks.
10. The method of claim 1 , wherein the C1 esterase inhibitor is purified from plasma.
11. The method of claim 1 , wherein the C1 esterase inhibitor is recombinantly produced.
12. The method of claim 1 , wherein the C1 esterase inhibitor comprises the amino acid sequence of residues 23 to 500 of SEQ ID NO:1.
13. The method of claim 1 , wherein the composition is a liquid formulation.
14. The method of claim 1 , wherein the composition is reconstituted from a lyophilized powder.
15. The method of claim 1 , wherein the administration of the composition comprising the C1 esterase inhibitor increases the level of C1 esterase inhibitor in the blood of the subject up to about 1 U/mL.
16. The method of claim 15 , wherein the increased blood levels of C1 esterase inhibitor are maintained at least 50% of the time between administrations.
17. The method of claim 1 , wherein the buffer is citrate.
18. The method of claim 1 , wherein the buffer is phosphate.