콜산

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. ( 1.2 ) Limitations of use: The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to SEDs or PDs including Zellweger spectrum disorders have not been established. ( 1.3 ). 1.1 Bile Acid Synthesis Disorders Due to Single Enzyme Defects CHOLBAM is indicated for the treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). 1.2. Peroxisomal Disorders Including Zellweger Spectrum Disorders CHOLBAM is indicated for 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. 1.3. Limitations of Use The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to SEDs or PDs including Zellweger spectrum disorders have not been established.

None. None ( 4 )

Exacerbation of Liver Impairment: Monitor liver function. Discontinue CHOLBAM if liver function worsens while on treatment. ( 5.1 ) 5.1 Exacerbation of Liver Impairment In clinical trials, evidence of liver impairment was present before treatment with CHOLBAM in approximately 86% (44/51) of patients with bile acid synthesis disorders due to SEDs and in approximately 50% (14/28) of patients with PDs including Zellweger spectrum disorders. Five of the patients (3 SED and 2 PD) with liver impairment at baseline experienced worsening serum transaminases, elevated bilirubin values, or worsening cholestasis on liver biopsy following treatment. Five additional patients (2 SED and 3 PD) who did not have baseline cholestasis experienced exacerbation of their liver disease while on treatment. In patients with cirrhosis, cases of severe hepatotoxicity have also been observed following postmarket use of CHOLBAM. Exacerbation of liver impairment by CHOLBAM in these patients cannot be ruled out. Six patients with SEDs underwent liver transplant, including four patients diagnosed with AKR1D1 deficiency, one with 3β-HSD deficiency, and one with CYP7A1 deficiency. Concurrent elevations of serum GGT and ALT may indicate CHOLBAM overdose [see Dosage and Administration ( 2.2 ) and Overdosage ( 10 )] . Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Discontinue treatment with CHOLBAM at any time if there are clinical or …

8.1 Pregnancy Pregnancy Surveillance Program There is a pregnancy surveillance program that monitors pregnancy outcomes in women exposed to CHOLBAM during pregnancy. Women who become pregnant during CHOLBAM treatment are encouraged to enroll. Patients or their health care provider should call Mirum Medical Information 1-855-676-4968. Risk Summary No studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM. Limited published case reports discuss pregnancies in women taking cholic acid for 3β-HSD deficiency resulting in healthy infants. These reports may not adequately inform the presence or absence of drug-associated risk with the use of CHOLBAM during …