Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication CHOLBAM is a bile acid indicated for: • Treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). ( 1.1 ) • Adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat-soluble vitamin absorption. FDA label
Also known as Cholic, Cholic Acid Capsules
Cholic acid · 2 trials · 2 indications
Concentration of serum alanine transaminase (ALT) and aspartate transaminase (AST)
Concentration of bile acids in serum (S) and urine (U). (abbreviations: chol.=cholenoic; monohydro=monohydroxy; dihydro=monohydro)
The level of atypical urinary bile acid secretion was scored using a scale of: 0, normal; 1, slight; 2, significant; or 3, marked. A Cochran-Mantel-Haenszel (CMH) test with modified ridit scoring was used to compare the difference between the score at baseline and the worst post-baseline score during treatment with cholic acid in this single-arm trial.
| Arm | Type | Description |
|---|---|---|
| Cholic Acid Capsule | EXPERIMENTAL | Manufactured cholic acid capsules |
| Cholic Acid | OTHER | Active drug |
| Name | Type | Description |
|---|---|---|
| Cholic acid | DRUG | The IUPAC name for cholic acid is 3 alpha,7alpha,12 alpha-trihydroxy-5 beta-cholanoic acid. The international nonproprietary name (INN) is cholic acid. Each patient will be given a box containing a 1 month supply of study drug. Each bottle will contain 90 capsules; each capsule will contain either 50 or 250 mg of manufactured cholic acid depending upon the child's weight. The study drug will be taken orally, in divided doses (as determined by the investigator), for a total daily dose of 10-15 mg/kg body weight. Parents of infants and young children who are unable to swallow the TBM cholic acid capsule will be instructed to sprinkle the contents of the capsule over 1-2 teaspoons of plain applesauce and feed it to the child. |
Inclusion Criteria: * must have stable transaminase levels within 2 times the upper limits of the normal range. * must have a diagnosis of an inborn error of bile acid synthesis. * must have signed the written informed consent/assent document before study start. * must be currently receiving curren...
Cholic acid is used to treat inborn errors of bile acid synthesis, a group of rare inherited disorders in which the body cannot produce normal bile acids. It is a small molecule therapy developed by Mirum Pharmaceuticals for these bile acid synthesis defects, which fall within the rare disease therapeutic area.
Cholic acid is developed by Mirum Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker MIRM. The company is the sponsor of the clinical program studying cholic acid in inborn errors of bile acid synthesis and bile acid synthesis defects.
Cholic acid is in Phase 3 clinical development for inborn errors of bile acid synthesis. Its Phase 3 program includes completed studies, and the drug is investigational rather than approved. Development has focused on pediatric and other patients with these rare inherited bile acid synthesis defects.
Cholic acid has been studied in Phase 3 trials including NCT01438411, an open label continuation study in subjects with inborn errors of bile acid synthesis that enrolled 53 participants in the United States, and NCT01115582, which evaluated cholic acid capsules in children with inborn errors of bile acid synthesis and enrolled 16 participants. Both trials are completed.
Cholic acid is a small molecule bile acid. In inborn errors of bile acid synthesis, patients cannot make normal bile acids, and cholic acid therapy is used to address that deficiency. It is being developed for bile acid synthesis defect and inborn errors of bile acid synthesis.
The cholic acid Phase 3 trials were uncontrolled, non-randomized studies. NCT01438411 was an open label continuation study, and NCT01115582 evaluated cholic acid capsules in children. Neither trial used randomization or a double-blind design, and both are now completed.