Tetrathiomolybdic acid for use in treating copper metabolism-associated diseases or disorders (wilson disease)
AssigneeAlexion Pharmaceuticals, Inc.
InventorsEugene Scott Swenson, Jennifer Lee Pocoski, Megan Teynor, Liliana-Georgiana Wegmann, Brian Meltzer, Barbara Davies
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Abstract
This disclosure relates to methods of treating a copper metabolism-associated disease or disorder, such as Wilson disease (WD).
Claims150
Tetrathiomolybdic acid or a pharmaceutically acceptable salt thereof for treating a copper metabolism associated disease or disorder in a subject,
- wherein a therapeutically effective amount of tetrathiomolybdic acid or a pharmaceutically acceptable salt thereof is administered to the subject;
- wherein the subject is at least 12 years old;
- and
- wherein the subject has an incomplete response and/or is intolerant to a standard of care treatment.
The tetrathiomolybdic acid or a pharmaceutically acceptable salt thereof of any one of claims 79-81, wherein the therapeutically effective amount of bis-choline tetrathiomolybdate is in the range of about 15 mg to about 60 mg per day.
A method for treating a copper metabolism associated disease or disorder in a subject, the method comprising:
- administering to the subject a therapeutically effective amount of tetrathiomolybdic acid or a pharmaceutically acceptable salt thereof;
- wherein the subject is at least 12 years old;
- and
- wherein the subject has an incomplete response and/or is intolerant to a standard of care treatment.
The method of any one of claims 51-4494 wherein the therapeutically effective amount of tetrathiomolybdic acid or a pharmaceutically acceptable salt thereof is sufficient to improve one or more neurological symptoms of the subject as measured according to Unified Wilson Disease Rating Scale (UWDRS) Part II and/or Part III, the one or more neurological symptoms selected from the group consisting of arising from a chair, arm and hand dystonia, cervical dystonia, chorea, facial expression, finger to nose, finger taps, gait, handwriting, head tremor, involuntary crying, jaw tremor, leg agility, oculomotor, postural arm tremor, postural leg tremor, posture, pyramidal signs, RAM of hands, rigidity, speech, stereotyped actions, tremor at rest, and wing-beating tremor.
A use of tetrathiomolybdic acid or a pharmaceutically acceptable salt thereof for the manufacture of a medicament for treating a copper metabolism associated disease or disorder in a subject,
- wherein a therapeutically effective amount of tetrathiomolybdic acid or a pharmaceutically acceptable salt thereof is administered to the subject;
- wherein the subject is at least 12 years old;
- and
- wherein the subject has an incomplete response and/or is intolerant to a standard of care treatment.
Dependencies are inferred from the claim text.