{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,11]],"date-time":"2026-07-11T20:34:18Z","timestamp":1783802058910,"version":"3.55.0"},"reference-count":23,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,5,2]],"date-time":"2024-05-02T00:00:00Z","timestamp":1714608000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clin Pharma and Therapeutics"],"published-print":{"date-parts":[[2024,9]]},"abstract":"<jats:p>On June 6, 2022, the FDA expanded the indications for mycophenolate mofetil (MMF) to include the prophylaxis of organ rejection in combination with other immunosuppressants in pediatric recipients of allogeneic heart or liver transplants aged 3\u2009months and older. The approved oral dosing regimen for these patients was a starting dose of 600\u2009mg\/m<jats:sup>2<\/jats:sup> with titration up to a maximum of 900\u2009mg\/m<jats:sup>2<\/jats:sup> twice daily. Data to support efficacy in pediatric patients were derived from established pharmacokinetic (PK) relationships across approved populations, a PK study in pediatric liver transplant recipients, and information from the Scientific Registry of Transplant Recipients database. Information supporting safety was based on comparing mycophenolic acid (MPA) exposure with that in pediatric kidney transplant recipients, the published literature, and post\u2010marketing safety reports. Efficacy in pediatric patients was established based on extrapolation of efficacy from studies in adult liver, adult heart, and pediatric kidney transplant populations, and similarity in MPA exposure between pediatric and adult patients. Review of the data supported an oral dosing regimen for pediatric heart transplant and liver transplant recipients consisting of a starting dose of 600\u2009mg\/m<jats:sup>2<\/jats:sup> up to a maximum of 900\u2009mg\/m<jats:sup>2<\/jats:sup> b.i.d. A dosage range for MMF is recommended recognizing that the MMF dose may be modified in clinical practice for myriad factors. The dosage recommendations in the labeling for pediatric liver and pediatric heart transplant patients are intended to permit individualized dosing based on clinical assessment of these factors.<\/jats:p>","DOI":"10.1002\/cpt.3288","type":"journal-article","created":{"date-parts":[[2024,5,2]],"date-time":"2024-05-02T12:23:08Z","timestamp":1714652588000},"page":"807-813","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Approval of Mycophenolate Mofetil for Prophylaxis of Organ Rejection in Pediatric Recipients of Heart or Liver Transplants: A Regulatory Perspective"],"prefix":"10.1002","volume":"116","author":[{"given":"Amer","family":"Al\u2010Khouja","sequence":"first","affiliation":[{"name":"Division of Inflammation and Immune Pharmacology, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research U.S. Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nadia","family":"Chaudhri","sequence":"additional","affiliation":[{"name":"Division of Rheumatology and Transplant Medicine, Office of New Drugs, Center for Drug Evaluation and Research U.S. Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1651-9961","authenticated-orcid":false,"given":"Ergun","family":"Velidedeoglu","sequence":"additional","affiliation":[{"name":"Division of Rheumatology and Transplant Medicine, Office of New Drugs, Center for Drug Evaluation and Research U.S. Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ozlem","family":"Belen","sequence":"additional","affiliation":[{"name":"Division of Rheumatology and Transplant Medicine, Office of New Drugs, Center for Drug Evaluation and Research U.S. Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Youwei","family":"Bi","sequence":"additional","affiliation":[{"name":"Division of Pharmacometrics, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research U.S. Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suresh","family":"Doddapaneni","sequence":"additional","affiliation":[{"name":"Division of Inflammation and Immune Pharmacology, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research U.S. Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0004-7585-0701","authenticated-orcid":false,"given":"Jianmeng","family":"Chen","sequence":"additional","affiliation":[{"name":"Division of Inflammation and Immune Pharmacology, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research U.S. Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,5,2]]},"reference":[{"key":"e_1_2_11_1_1","doi-asserted-by":"publisher","DOI":"10.1111\/ajt.16494"},{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/ajt.16492"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1399-3062.2009.00407.x"},{"key":"e_1_2_11_4_1","unstructured":"Genentech Inc.CellCept (mycophenolate mofetil) [package insert]. 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