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CymaBay Initiates IDEAL, a Phase 3 Placebo-Controlled, Randomized Study of Seladelpar
Clinical Trials, PBC News  |   August 14, 2023

CymaBay Initiates IDEAL, a Phase 3 Placebo-Controlled, Randomized Study of Seladelpar

The 52-week IDEAL study aims to enroll 75 patients with PBC who have an incomplete response or intolerance to ursodeoxycholic acid (UDCA), in each case with ALP greater than the
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Ipsen and GENFIT announce positive Phase 3 trial data
Clinical Trials, PBC News  |   June 30, 2023

Ipsen and GENFIT announce positive Phase 3 trial data

Ipsen and GENFIT announce positive topline data from the pivotal ELATIVE Phase III trial. In the trial the efficacy and safety of elafibranor, an investigational dual α,δ PPAR agonist, is
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Fixed dose combination OCA/Bezafibrate positive interim Phase 2 study results.
Clinical Trials, PBC News  |   June 11, 2023

Fixed dose combination OCA/Bezafibrate positive interim Phase 2 study results.

Intercept Pharmaceuticals announces new data showing potential of Fixed-Dose Combination of OCA and Bezafibrate to normalize multiple biomarkers in PBC to be featured in podium presentation at EASL Congress June
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Linerixibat GLISTEN phase 3 study now enrolling
Clinical Trials, PBC News  |   May 31, 2023

Linerixibat GLISTEN phase 3 study now enrolling

The GLISTEN clinical trial is investigating a potential new treatment specifically for PBC itch and is now enrolling patients worldwide.  In Canada, the study is currently enrolling in Toronto, Ontario.
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Fixed-dose combination OCA/Bezafibrate granted orphan drug designation by FDA
Clinical Trials, PBC News  |   May 18, 2023

Fixed-dose combination OCA/Bezafibrate granted orphan drug designation by FDA

Intercept Pharmaceuticals is investigating a fixed-dose combination of OCA and bezafibrate for the potential treatment of individuals with PBC. Clinical trials are underway to establish safety and efficacy. OCA, a
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Pacific Study Phase 2 now enrolling in Canada
Clinical Trials, PBC News  |   March 1, 2023

Pacific Study Phase 2 now enrolling in Canada

The Phase 2 PACIFIC study will enroll approximately 58 patients with moderate-to-severe pruritus due to PBC or PSC.   Now enrolling in Canada, see what’s involved here. The study comprises a
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Clinical Trials, PBC News  |   February 18, 2023

Evidence of efficacy of OCA for PBC patients

A study published in the Gastroenterology (September 2022) showed that people receiving OCA for primary biliary cholangitis (PBC) in a clinical trial setting had greater transplant-free survival to patients with
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Clinical Trials, PBC News  |   August 19, 2022

Seladelpar – Phase 2 results published

“This study demonstrates the significant anticholestatic effects of seladelpar in patients with PBC who had suboptimally responded to UDCA or were UDCA intolerant. Evidence of seladelpar’s dose-dependent efficacy, safety, and
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Clinical Trials, PBC News  |   May 24, 2022

Clinical studies of Seladelpar – new analysis

New analyses of clinical studies of Seladelpar were presented during Digestive Disease Week® (DDW) in San Diego on May 23, 2022.   The three presentations were: “Seladelpar Treatment of Patients With
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Setanaxib TRANSFORM study now enrolling
Clinical Trials, PBC News  |   April 7, 2022

Setanaxib TRANSFORM study now enrolling

The purpose of this clinical research study is to evaluate the effects of an investigational product (setanaxib) as a potential additional treatment for PBC. The study is now enrolling in
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9/10 patients with PBC are women 35-60 years old

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Initial assessment
• Ultrasound should be used to exclude other causes of cholestasis.
• Physical examination should be used to screen for hepatomegaly, splenomegaly and extrahepatic signs
of advanced liver disease.

Unexplained cholestasis
• Elevated ALP but AMA-negative (<1/40)
• Concern over, or presence of, autoimmune hepatitis

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Additional assessments

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If further assessments confirm PBC

Confirmed PBC

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*Liver biopsy is rarely required for diagnosis in the correct clinical context; consider if disease-specific autoantibodies are absent, or concern over features of AIH or coexistent NAFLD.

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Disease management
Pruritus, fatigue, sicca complex, bone density, coexistent autoimmune disease, CV risk and metabolic syndrome

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Assess pre-treatment disease stage and long-term risk of disease progression
• The strongest risk factors for inadequate response to UDCA are early age at diagnosis and advanced disease stage at presentation. Other risk factors also exist for inadequate response to UDCA, including male gender (associated with more advanced disease at presentation).
• Patients with an inadequate response to UDCA, and those with cirrhosis, are at the greatest risk of disease progression and complications from PBC.
• Severity of symptoms does not necessarily correlate with disease stage in PBC, however, severe pruritus can indicate an aggressively ductopenic variant of PBC, which is associated with a poor prognosis.

 

Hirschfield et al. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. J Hepatol
2017; 67(1): 145–172
Zhang et al. Early biochemical response to ursodeoxycholic acid and long-term prognosis of primary biliary cirrhosis:
results of a 14-year cohort study. Hepatology 2013; 58(1): 264–272 3. Corpechot et al. Noninvasive elastography-based assessment of liver
fibrosis progression and prognosis in primary biliary cirrhosis. Hepatology 2012; 56(1): 198–208

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Assess 1st line treatment response within 6–12 months
• Most biochemical response criteria have been validated as predicting long-term disease progression at 12 months from UDCA initiation.1 A 12-month period is conventionally used to assess biochemical response to UDCA, but it has been demonstrated that evaluation at 6 months may be equally discriminatory.
• Elastography should be used yearly to assess liver fibrosis. Elastography has been shown as one of the best surrogate markers for the detection of cirrhosis or severe fibrosis.1 It has also been demonstrated that liver stiffness measurements (as measured by elastography) of greater than 9.6kPa are associated with a 5-fold increased risk of liver decompensation, transplantation or death.3 However, the ability of elastography to predict disease progression in PBC requires further validation.

 

Hirschfield et al. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. J Hepatol
2017; 67(1): 145–172
Zhang et al. Early biochemical response to ursodeoxycholic acid and long-term prognosis of primary biliary cirrhosis:
results of a 14-year cohort study. Hepatology 2013; 58(1): 264–272 3. Corpechot et al. Noninvasive elastography-based assessment of liver
fibrosis progression and prognosis in primary biliary cirrhosis. Hepatology 2012; 56(1): 198–208

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Abbreviated Prescribing Information
Presentation: OCALIVA supplied as film-coated tablets containing 5 mg and 10 mg obeticholic acid.
Indication: For the treatment of primary biliary cholangitis (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.
Dosage and administration: Oral administration. Hepatic status must be known before initiating treatment. In patients with normal or mildly impaired (Child Pugh Class A) hepatic function, the starting dose is 5 mg once daily. Based on an assessment of tolerability after 6 months, the dose should be increased to 10 mg once daily if adequate reduction of alkaline phosphatase (ALP) and/or total bilirubin have not been achieved. No dose adjustment of concomitant UDCA is required in patients receiving obeticholic acid. For cases of severe pruritus, dose management includes reduction, temporal interruption or discontinuation for persistent intolerable pruritus; use of bile acid binding agents or antihistamines (see SmPC).
Moderate to severe hepatic impairment: In patients with Child Pugh B or C hepatic impairment, a reduced starting dose of 5 mg once weekly is required. After 3 months, depending on response and tolerability, the starting dose may be titrated to 5 mg twice weekly and subsequently to 10 mg twice weekly (at least 3 days between doses) if adequate reductions in ALP and/or total bilirubin have not been achieved. No dose adjustment required in Child Pugh Class A function.
Mild or moderate renal impairment: No dose adjustments are required. Paediatric population: No data. Elderly: No dose adjustment required; limited data exist. Contraindications: Hypersensitivity to the active substance or any excipients. Complete biliary obstruction. Special warnings and precautions for use: After initiation, patients should be monitored for progression of PBC with frequent clinical and laboratory assessment of those at increased risk of hepatic decompensation. Dose frequency should be reduced in patients who progress from Child Pugh A to Child Pugh B or C Class disease. Serious liver injury and death have been reported in patients with moderate/severe impairment who did not receive appropriate dose reduction. Liver-related adverse events have been observed within the first month of treatment and have included elevations in alanine amino transferase (ALT), aspartate aminotransferase (AST) and hepatic decompensation.
Interactions: Following co-administration of warfarin and obeticholic acid, International Normalised Ratio (INR) should be monitored and the dose of warfarin adjusted, if needed, to maintain the target INR range. Therapeutic monitoring of CYP1A2 substrates with narrow therapeutic index (e.g. theophylline and tizanidine) is recommended. Obeticholic acid should be taken at least 4-6 hours before or after taking a bile acid binding resin, or at as great an interval as possible. Fertility, pregnancy and lactation: Avoid use in pregnancy. Either discontinue breast-feeding or discontinue/abstain from obeticholic acid therapy taking into account the benefit of breast-feeding for the child and the benefit of therapy for the woman. No clinical data on fertility effects. Undesirable effects: Very common (≥1/10) adverse reactions were pruritus, fatigue, and abdominal pain and discomfort. The most common adverse reaction leading to discontinuation was pruritus. The majority of pruritus occurred within the first month of treatment and tended to resolve over time with continued dosing. Other commonly (≥1/100 to <1/10) reported adverse reactions are, thyroid function abnormality, dizziness, palpitations, oropharyngeal pain, constipation, eczema, rash, arthralgia, peripheral oedema, and pyrexia. Please refer to the SmPC for a full list of undesirable effects.
Overdose: Liver-related adverse reactions were reported with higher than recommended doses of obeticholic acid. Patients should be carefully observed, and supportive care administered, as appropriate. Legal category: POM Marketing authorisation numbers: EU/1/16/1139/001 & 002
Marketing authorisation holder: Intercept Pharma Ltd, 2 Pancras Square, London, N1C 4AG, United Kingdom Package quantities and basic NHS cost: OCALIVA 5 mg and 10 mg £2,384.04 per bottle of 30 tablets. Date of revision: 11th April 2018

 

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