Compositions and their use for the treatment of A1-antitrypsin deficiency
The invention relates to pharmaceutical compositions comprising (S)-1-(2-(trifluoromethyl)phenyl)sulfonyl)piperidine-3-carboxylic acid and their medical use, for example for use in the treatment of α 1 -antitrypsin deficiency (AATD).
1 . A method of treating a disease or disorder characterised by low plasma levels of A1AT in a human subject, comprising the step of administering a pharmaceutical composition comprising(S)-1-((2-(trifluoromethyl)phenyl)sulfonyl)piperidine-3-carboxylic acid
or a pharmaceutically acceptable salt form,
and a pharmaceutically or therapeutically acceptable excipient or carrier, at a daily dose of about 1-6 mg/kg.
2 . The method of claim 1 , in which the disease or disorder is a1-antitrypsin deficiency (AATD).
3 . The method of claim 1 , wherein the administering to the human subject is at a daily dose of about 2-5 mg/kg.
4 . The method of claim 3 , wherein the daily dose is about 2 mg/kg.
5 . The method of claim 3 , wherein the daily dose is about 3 mg/kg.
6 . The method of claim 3 , wherein the daily dose is about 4 mg/kg.
7 . The method of claim 3 , wherein the daily dose is about 5 mg/kg.
8 . The method of claim 1 , wherein the composition is administered in oral form.
9 . The method of claim 1 , wherein the composition is administered intravenously.