Cholic Acid to Treat HSD3B7 and AKR1D1 Deficiencies

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To the Editor: We read Alan Hofmann's editorial (1) concerning the article by Jahnel et al (2). We feel that additional information deserve to be brought to the Journal of Pediatric Gastroenterology and Nutrition readership knowledge. The sentence “one wonders whether desiccated bovine bile … would also be effective.” (1) sounds like an option from times long gone, which is no longer defendable in the 21st century, with law, regulation, and patient protection at such high level of complexity to deal with. The readership should know that not 1 but 2 laboratories have a marketing authorization and provide cholic acid therapy for 3β-hydroxy-Δ5-C27-steroid dehydrogenase and Δ4–3-oxosteroid-5β-reductase deficiencies (Cholbam marketed by Retrophin [United States] and Orphacol marketed by CTRS [Europe]) (3,4). In the editorial, only Cholbam and Retrophin are mentioned and it is written that Dr Hofmann serves as a consultant for Retrophin (1). In Jahnel's article, no company and commercial drug names are mentioned (2). Considering Dr Hofmann editorial and for transparency reasons, we feel that the Journal of Pediatric Gastroenterology and Nutrition readership should be aware that depending on companies, cholic acid is sold at quite different costs/year (50-mg tablet annual supply, 50 mg/day): 98,000 US$ for Cholbam/Retrophin in the United States versus 31,300 US$ for Orphacol/CTRS in France.
Beyond our own conflicts of interest (2) and because Cholbam has been mentioned in the editorial (1), we believe that mentioning Orphacol is all the more important that the survey has been conducted in Europe, where Orphacol is the only EMA-approved drug to treat these diseases (4).
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