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Cholic acid
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<h2 id="medical-uses">Medical uses</h2> <p>Cholic acid, sold under the brand name Cholbam, is approved for use in the United States and is indicated as a treatment for children and adults with bile acid synthesis disorders due to single enzyme defects, and for peroxisomal disorders such as <a href="/facts/Zellweger_syndrome/RJVhq1by">Zellweger syndrome</a>.<a class="footnote-ref" id="fnref:7" href="#fn:7"><sup>7</sup></a><a class="footnote-ref" id="fnref:8" href="#fn:8"><sup>8</sup></a><a class="footnote-ref" id="fnref:9" href="#fn:9"><sup>9</sup></a> </p><p>It was approved for use in the European Union in September 2013, and is sold under the brand name Orphacol.<a class="footnote-ref" id="fnref:10" href="#fn:10"><sup>10</sup></a> It is indicated for the treatment of inborn errors in primary bile-acid synthesis due to <a href="/facts/3%25CE%25B2-hydroxy-%25CE%25945-C27-steroid_oxidoreductase/UTLRwGCI">3β-hydroxy-Δ5-C27-steroid oxidoreductase</a> deficiency or <a href="/facts/%25CE%25944-3-oxosteroid-5%25CE%25B2-reductase/lh36at1h">Δ4-3-oxosteroid-5β-reductase</a> deficiency in infants, children and adolescents aged one month to 18 years and adults.<a class="footnote-ref" id="fnref:11" href="#fn:11"><sup>11</sup></a> </p><p>Cholic acid FGK (Kolbam) was approved for medical use in the European Union in November 2015.<a class="footnote-ref" id="fnref:12" href="#fn:12"><sup>12</sup></a> It is indicated for the treatment of inborn errors of primary bile acid synthesis, in infants from one month of age for continuous lifelong treatment through adulthood, encompassing the following single enzyme defects:<a class="footnote-ref" id="fnref:13" href="#fn:13"><sup>13</sup></a> </p> <ul><li><a href="/facts/Sterol_27-hydroxylase/NBfccTy4">sterol 27-hydroxylase</a> deficiency (presenting as <a href="/facts/Cerebrotendinous_xanthomatosis/ERSsqfmQ">cerebrotendinous xanthomatosis</a>, CTX);<a class="footnote-ref" id="fnref:14" href="#fn:14"><sup>14</sup></a></li> <li><a href="/facts/Alpha-methylacyl-CoA_racemase/kDoSRlmI">2- (or alpha-) methylacyl-CoA racemase</a> (AMACR) deficiency;<a class="footnote-ref" id="fnref:15" href="#fn:15"><sup>15</sup></a></li> <li>cholesterol 7 alpha-hydroxylase (CYP7A1) deficiency.<a class="footnote-ref" id="fnref:16" href="#fn:16"><sup>16</sup></a></li></ul> <p>The most common side effects include peripheral neuropathy (nerve damage in the hands and feet), diarrhea, nausea (feeling sick), acid reflux (stomach acid flowing up into the mouth), esophagitis (inflammation of the food pipe), jaundice (yellowing of the skin and eyes), skin problems (lesions) and malaise (feeling unwell).<a class="footnote-ref" id="fnref:17" href="#fn:17"><sup>17</sup></a> </p> <h2 id="cancer">Cancer</h2> <p>A <a href="/facts/Meta-analysis/IcfChd8z">meta-analysis</a> on the relationship of fecal <a href="/facts/Bile_acid/ojTJzf0c">bile acid</a> concentrations to the development and progression of <a href="/facts/Colorectal_cancer/nV592sW7">colorectal cancer</a> was reported in 2025.<a class="footnote-ref" id="fnref:18" href="#fn:18"><sup>18</sup></a> It was found that higher fecal concentrations of cholic acid are associated with a higher risk/incidence of colorectal cancer.<a class="footnote-ref" id="fnref:19" href="#fn:19"><sup>19</sup></a> Bile acids have been implicated as <a href="/facts/Carcinogen/soH8Y9CE">carcinogens</a> in the <a href="/facts/Gastrointestinal_tract/nK2zwqBS">gastrointestinal tract</a>.<a class="footnote-ref" id="fnref:20" href="#fn:20"><sup>20</sup></a> </p> <h2 id="interactive-pathway-map">Interactive pathway map</h2> <p><i>Click on genes, proteins and metabolites below to link to respective articles.</i> <a class="footnote-ref" id="fnref:21" href="#fn:21"><sup>21</sup></a> </p> <h2 id="references">References</h2> <ol> <li id="fn:1"><p>Colleen Smith; Lieberman, Michael; Marks, Dawn B.; Allan D. Marks (2007). Marks' essential medical biochemistry. Hagerstwon, MD: Lippincott Williams & Wilkins. ISBN 978-0-7817-9340-7. <a href="978-0-7817-9340-7" target="_blank">978-0-7817-9340-7</a> <a href="#fnref:1" class="footnote-back-ref">↩</a></p></li> <li id="fn:2"><p>Bennion LJ, Ginsberg RL, Gernick MB, Bennett PH (January 1976). "Effects of oral contraceptives on the gallbladder bile of normal women". N. Engl. J. Med. 294 (4): 189–92. doi:10.1056/NEJM197601222940403. PMID 1244533. <a href="/wiki/Doi_(identifier)" target="_blank">/wiki/Doi_(identifier)</a> <a href="#fnref:2" class="footnote-back-ref">↩</a></p></li> <li id="fn:3"><p>Chiang JY (October 2009). "Bile acids: regulation of synthesis". Journal of Lipid Research. 50 (10): 1955–66. doi:10.1194/jlr.R900010-JLR200. PMC 2739756. PMID 19346330. <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2739756" target="_blank">https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2739756</a> <a href="#fnref:3" class="footnote-back-ref">↩</a></p></li> <li id="fn:4"><p>Iser JH, Dowling H, Mok HY, Bell GD (August 1975). "Chenodeoxycholic acid treatment of gallstones. A follow-up report and analysis of factors influencing response to therapy". The New England Journal of Medicine. 293 (8): 378–83. doi:10.1056/NEJM197508212930804. PMID 1152936. <a href="/wiki/Doi_(identifier)" target="_blank">/wiki/Doi_(identifier)</a> <a href="#fnref:4" class="footnote-back-ref">↩</a></p></li> <li id="fn:5"><p>Alan F. Hofmann; Johnson L. Thistle; Peter D. Klein; Patricia A. Szczepanik; Paulina Y. S. Yu (1978). "Chenotherapy for Gallstone Dissolution, II. Induced Changes in Bile Composition and Gallstone Response". JAMA. 239 (12): 1138–1144. doi:10.1001/jama.1978.03280390034017. PMID 628065. <a href="/wiki/Doi_(identifier)" target="_blank">/wiki/Doi_(identifier)</a> <a href="#fnref:5" class="footnote-back-ref">↩</a></p></li> <li id="fn:6"><p>Hofmann AF, Hagey LR, Krasowski MD (February 2010). "Bile salts of vertebrates: structural variation and possible evolutionary significance". J. Lipid Res. 51 (2): 226–46. doi:10.1194/jlr.R000042. PMC 2803226. PMID 19638645. <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2803226" target="_blank">https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2803226</a> <a href="#fnref:6" class="footnote-back-ref">↩</a></p></li> <li id="fn:7"><p>"Cholbam (cholic acid) Capsules". U.S. Food and Drug Administration (FDA). 13 April 2015. Retrieved 3 May 2020. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/nda/2015/205750Orig1s000TOC.cfm" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/nda/2015/205750Orig1s000TOC.cfm</a> <a href="#fnref:7" class="footnote-back-ref">↩</a></p></li> <li id="fn:8"><p>"Cholbam- cholic acid capsule". DailyMed. 12 July 2019. Retrieved 2 May 2020. <a href="https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e5b67402-8550-4604-97a0-c7b149fbf753" target="_blank">https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e5b67402-8550-4604-97a0-c7b149fbf753</a> <a href="#fnref:8" class="footnote-back-ref">↩</a></p></li> <li id="fn:9"><p>"FDA approves Cholbam to treat rare bile acid synthesis disorders". U.S. Food and Drug Administration (FDA) (Press release). 17 March 2015. Archived from the original on 26 January 2018. Retrieved 19 May 2020. This article incorporates text from this source, which is in the public domain. <a href="https://web.archive.org/web/20180126023504/https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm438572.htm" target="_blank">https://web.archive.org/web/20180126023504/https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm438572.htm</a> <a href="#fnref:9" class="footnote-back-ref">↩</a></p></li> <li id="fn:10"><p>"Orphacol EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 2 May 2020. Text was copied from this source which is under copyright of the European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/orphacol" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/orphacol</a> <a href="#fnref:10" class="footnote-back-ref">↩</a></p></li> <li id="fn:11"><p>"Orphacol EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 2 May 2020. Text was copied from this source which is under copyright of the European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/orphacol" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/orphacol</a> <a href="#fnref:11" class="footnote-back-ref">↩</a></p></li> <li id="fn:12"><p>"Kolbam EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 19 May 2020. Text was copied from this source which is under copyright of the European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam</a> <a href="#fnref:12" class="footnote-back-ref">↩</a></p></li> <li id="fn:13"><p>"Kolbam EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 19 May 2020. Text was copied from this source which is under copyright of the European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam</a> <a href="#fnref:13" class="footnote-back-ref">↩</a></p></li> <li id="fn:14"><p>"Kolbam EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 19 May 2020. Text was copied from this source which is under copyright of the European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam</a> <a href="#fnref:14" class="footnote-back-ref">↩</a></p></li> <li id="fn:15"><p>"Kolbam EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 19 May 2020. Text was copied from this source which is under copyright of the European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam</a> <a href="#fnref:15" class="footnote-back-ref">↩</a></p></li> <li id="fn:16"><p>"Kolbam EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 19 May 2020. Text was copied from this source which is under copyright of the European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam</a> <a href="#fnref:16" class="footnote-back-ref">↩</a></p></li> <li id="fn:17"><p>"Kolbam EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 19 May 2020. Text was copied from this source which is under copyright of the European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/kolbam</a> <a href="#fnref:17" class="footnote-back-ref">↩</a></p></li> <li id="fn:18"><p>Yang S, Wang Y, Sheng L, Cui W, Ma C (January 2025). "The effect of fecal bile acids on the incidence and risk-stratification of colorectal cancer: an updated systematic review and meta-analysis". Sci Rep. 15 (1): 740. doi:10.1038/s41598-024-84801-6. PMC 11698987. PMID 39753873. <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11698987" target="_blank">https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11698987</a> <a href="#fnref:18" class="footnote-back-ref">↩</a></p></li> <li id="fn:19"><p>Yang S, Wang Y, Sheng L, Cui W, Ma C (January 2025). "The effect of fecal bile acids on the incidence and risk-stratification of colorectal cancer: an updated systematic review and meta-analysis". Sci Rep. 15 (1): 740. doi:10.1038/s41598-024-84801-6. PMC 11698987. PMID 39753873. <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11698987" target="_blank">https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11698987</a> <a href="#fnref:19" class="footnote-back-ref">↩</a></p></li> <li id="fn:20"><p>Bernstein H, Bernstein C (January 2023). "Bile acids as carcinogens in the colon and at other sites in the gastrointestinal system". Exp Biol Med (Maywood). 248 (1): 79–89. doi:10.1177/15353702221131858. PMC 9989147. PMID 36408538. <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9989147" target="_blank">https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9989147</a> <a href="#fnref:20" class="footnote-back-ref">↩</a></p></li> <li id="fn:21"><p>The interactive pathway map can be edited at WikiPathways: "Statin_Pathway_WP430". <a href="http://www.wikipathways.org/index.php/Pathway:WP430" target="_blank">http://www.wikipathways.org/index.php/Pathway:WP430</a> <a href="#fnref:21" class="footnote-back-ref">↩</a></p></li> </ol>