AI Drug Discovery for Pharma and Biotech

Drug discovery

4

drugs

With orphan designations

Overview

Isolated biliary atresia (IBA) is a progressive neonatal cholangiopathy causing obliteration of intra-/extrahepatic bile ducts, presenting with persistent jaundice, acholic stools, and conjugated hyperbilirubinemia. Etiology involves genetic, immune, and environmental interactions, though non-hereditary. Diagnosis requires cholangiography confirmation after excluding other cholestatic causes. Kasai portoenterostomy remains first-line therapy, but 40-60% require liver transplantation by age 2 without sustained bile flow [1][3][5].

Population

  • Birth prevalence: 1/15,000-19,000 in Europe/North America [1], ~0.65/10,000 in U.S. studies [2][17]

  • Higher incidence in females (1.68:1 ratio) and preterm infants [2][10]

  • Potential geographic variation with rural areas showing increased incidence [10][14]

Burden

  • Leading pediatric indication for liver transplantation worldwide [5][15]

  • Complications: Cholangitis (40-60%), portal hypertension (50-70%), progressive cirrhosis [1][3][7]

  • Healthcare costs: Prolonged hospitalization, lifelong monitoring, and transplant-related expenses [5][15]

Therapies

  • Primary surgical: Kasai portoenterostomy (<70 days optimal), achieving native liver survival in 25-50% at 10 years [1][3][15]

  • Adjuvant medical: Limited efficacy of corticosteroids/IVIG; nutritional support (vitamin K, ursodiol) and infection prophylaxis [7][11]

  • Transplant rescue: 50% require liver transplantation by age 2, with 85% 10-year post-transplant survival [3][5]

Categories: rare abdominal surgical diseases, rare developmental anomalies during embryogenesis, rare hepatic diseases, rare transplant-related disorders

Research Papers

1,563 drug discovery papers about Isolated biliary atresia, with 2 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

1,563 drug discovery papers about Isolated biliary atresia, with 2 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-08 | The bile acid-gut microbiota-vitamin D axis: new insights into biliary atresia.

Biliary atresia (BA) is a progressive fibroinflammatory disease affecting both intrahepatic and extrahepatic bile ducts. It leads to cholestasis, hepatic fibrosis, and eventually biliary cirrhosis. Although BA is the most common cause of neonatal cholestasis, its exact pathogenesis remains incompletely understood. Emerging evidence suggests that disturbances in bile acid metabolism, gut microbiota dysbiosis, and vitamin D deficiency may play critical roles in the initiation and progression of BA. This review systematically summarizes the dynamic evolution characteristics of bile acid profiles, gut microbiota composition, and vitamin D levels in children with BA across different disease stages. It further explores the potential positive feedback loop effects of the bile acid-gut microbiota-vitamin D axis in the pathogenesis and progression of the disease. Additionally, this review discusses potential diagnostic value and therapeutic strategies based on this axis. Interventions targeting bile acid metabolism, modulating the gut microbiota, and supplementing vitamin D have shown promise in altering the disease course. Despite these advances, further research is needed to elucidate the precise molecular mechanisms underlying this axis in BA. Such knowledge will pave the way for more effective preventive and therapeutic strategies for this complex disease.

Open article ↗



2026-07-06 | Mesenchymal stem cells in intrahepatic and extrahepatic biliary diseases: mechanisms and therapeutic applications.

Mesenchymal stem cells (MSCs) have emerged as promising therapeutic candidates for a wide range of intrahepatic and extrahepatic biliary diseases. However, their clinical application remains limited by an incomplete understanding of underlying mechanisms and heterogeneous therapeutic outcomes. This review provides a comprehensive overview of recent advances in MSC-based therapies for biliary system diseases, including primary biliary cholangitis, intrahepatic biliary fibrosis and cirrhosis, biliary atresia, primary sclerosing cholangitis, and cholangiocarcinoma. We summarize both preclinical and clinical evidence and focus on key therapeutic mechanisms, including immunomodulation, antifibrotic activity, regulation of bile acid metabolism, and protection of biliary epithelial cells.

Open article ↗



2026-06-18 | Antibiotic Prophylaxis Optimization Using Fecal Surveillance Culture in Operated Biliary Atresia Patients: A Retrospective Study on Cholangitis Prevention.

Antibiotics are central to preventing and treating cholangitis following Kasai portoenterostomy (KPE).There is an increasing evidence of resistance to commonly used antibiotics. Study explores an inexpensive and noninvasive method of monitoring and adapting antibiotic therapy post KPE using fecal surveillance cultures (FSCs).Primary aim was to assess load of antibiotic resistance among organisms isolated on FSC and the effect of choosing antibiotic based on this resistance pattern. One secondary aim was to assess the relationship between steroid initiation following KPE and cholangitis. It is a retrospective, observational study including 55 post-KPE patients. FSC were used to guide antibiotic prophylaxis and to tailor treatment during cholangitis episodes in select patients unresponsive to standard therapy. Post-KPE FSC was performed in 45.45% (25/55) patients, isolating Escherichia coli and Klebsiella pneumoniae, with notable resistance to cephalosporins, fluoroquinolones, and carbapenems; tigecycline showed the least resistance. During cholangitis episodes, FSC was done in 65.52% (19/29) cases, isolating E. coli, K. pneumoniae, Enterobacter cloacae, and Pseudomonas aeruginosa. Cephalosporin and fluoroquinolone resistance was highest followed by carbapenem and aminoglycoside with lowest being tigecycline resistance. Resistance was significantly higher in Klebsiella compared to E. coli (P < 0.05). Steroid initiation was significantly earlier in patients who developed cholangitis (15.27 ± 7.69 days post-KPE) compared to those who did not (21.47 ± 9.43 days; P = 0.019). Antibiotic prophylaxis post-KPE should be guided as per the FSC-sensitivity pattern as the rates of resistance to commonly used antibiotics is high. Caution is needed in initiating steroids very early after surgery.

Open article ↗



2026-07-08 | The bile acid-gut microbiota-vitamin D axis: new insights into biliary atresia.

Biliary atresia (BA) is a progressive fibroinflammatory disease affecting both intrahepatic and extrahepatic bile ducts. It leads to cholestasis, hepatic fibrosis, and eventually biliary cirrhosis. Although BA is the most common cause of neonatal cholestasis, its exact pathogenesis remains incompletely understood. Emerging evidence suggests that disturbances in bile acid metabolism, gut microbiota dysbiosis, and vitamin D deficiency may play critical roles in the initiation and progression of BA. This review systematically summarizes the dynamic evolution characteristics of bile acid profiles, gut microbiota composition, and vitamin D levels in children with BA across different disease stages. It further explores the potential positive feedback loop effects of the bile acid-gut microbiota-vitamin D axis in the pathogenesis and progression of the disease. Additionally, this review discusses potential diagnostic value and therapeutic strategies based on this axis. Interventions targeting bile acid metabolism, modulating the gut microbiota, and supplementing vitamin D have shown promise in altering the disease course. Despite these advances, further research is needed to elucidate the precise molecular mechanisms underlying this axis in BA. Such knowledge will pave the way for more effective preventive and therapeutic strategies for this complex disease.

Open article ↗



2026-07-06 | Mesenchymal stem cells in intrahepatic and extrahepatic biliary diseases: mechanisms and therapeutic applications.

Mesenchymal stem cells (MSCs) have emerged as promising therapeutic candidates for a wide range of intrahepatic and extrahepatic biliary diseases. However, their clinical application remains limited by an incomplete understanding of underlying mechanisms and heterogeneous therapeutic outcomes. This review provides a comprehensive overview of recent advances in MSC-based therapies for biliary system diseases, including primary biliary cholangitis, intrahepatic biliary fibrosis and cirrhosis, biliary atresia, primary sclerosing cholangitis, and cholangiocarcinoma. We summarize both preclinical and clinical evidence and focus on key therapeutic mechanisms, including immunomodulation, antifibrotic activity, regulation of bile acid metabolism, and protection of biliary epithelial cells.

Open article ↗



2026-06-18 | Antibiotic Prophylaxis Optimization Using Fecal Surveillance Culture in Operated Biliary Atresia Patients: A Retrospective Study on Cholangitis Prevention.

Antibiotics are central to preventing and treating cholangitis following Kasai portoenterostomy (KPE).There is an increasing evidence of resistance to commonly used antibiotics. Study explores an inexpensive and noninvasive method of monitoring and adapting antibiotic therapy post KPE using fecal surveillance cultures (FSCs).Primary aim was to assess load of antibiotic resistance among organisms isolated on FSC and the effect of choosing antibiotic based on this resistance pattern. One secondary aim was to assess the relationship between steroid initiation following KPE and cholangitis. It is a retrospective, observational study including 55 post-KPE patients. FSC were used to guide antibiotic prophylaxis and to tailor treatment during cholangitis episodes in select patients unresponsive to standard therapy. Post-KPE FSC was performed in 45.45% (25/55) patients, isolating Escherichia coli and Klebsiella pneumoniae, with notable resistance to cephalosporins, fluoroquinolones, and carbapenems; tigecycline showed the least resistance. During cholangitis episodes, FSC was done in 65.52% (19/29) cases, isolating E. coli, K. pneumoniae, Enterobacter cloacae, and Pseudomonas aeruginosa. Cephalosporin and fluoroquinolone resistance was highest followed by carbapenem and aminoglycoside with lowest being tigecycline resistance. Resistance was significantly higher in Klebsiella compared to E. coli (P < 0.05). Steroid initiation was significantly earlier in patients who developed cholangitis (15.27 ± 7.69 days post-KPE) compared to those who did not (21.47 ± 9.43 days; P = 0.019). Antibiotic prophylaxis post-KPE should be guided as per the FSC-sensitivity pattern as the rates of resistance to commonly used antibiotics is high. Caution is needed in initiating steroids very early after surgery.

Open article ↗



Access all drug discovery articles and probability of success in trials forecasts:

Access all drug discovery articles and probability of success in trials forecasts:

Drug Discovery Landscape

4 orphan drug designations for Isolated biliary atresia.

4 orphan drug designations for Isolated biliary atresia.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

maralixibat

small molecules

FDA

2020-10-21

Mirum Pharmaceuticals, Inc.

Maralixibat chloride

small molecules

EMA

2020-07-27

Mirum Pharmaceuticals International B.V.

odevixibat

small molecules

FDA

2019-01-16

Ipsen Biopharmaceuticals, Inc.

(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid

small molecules

EMA

2018-12-14

Ipsen Pharma

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At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.