Efficacy
The names of the extension studies that evaluated the long-term safety and efficacy of odevixibat in patients with PFIC and ALGS
What is PEDFIC2 and ASSERT-EXT?
The recommended starting dose of BYLVAY for PFIC and ALGS patients
What is 40 µg/kg per day for PFIC, and 120 µg/kg per day for ALGS
The most common side effect of BYLVAY in ASSERT
What is diarrhea?
The name of the registry formed to elucidate the natural history and long-term outcomes of PFIC
What is NAPPED?
IBAT inhibitors, such as odevixibat and maralixibat, work by blocking this process in the ileum to reduce circulating bile acids and pruritus
What is ileal bile acid reuptake/enterohepatic circulation of bile acids
Based on the data submitted and reviewed by Health Canada, the safety and efficacy of BYLVAY in pediatric patients aged __ months or older for the treatment of pruritus in ALGS have been established
What is 12?
The three ways Odevixibat can be administered, giving flexibility for children who have difficulty swallowing capsules
What is swallowed whole, opened and mixed into an age-appropriate liquid, or opened and mixed into soft food?
The dose of BYLVAY associated with a greater incidence of adverse events in PEDFIC
What is similar safety profiles were observed for both doses?
The GALA registry revealed that surgical biliary diversion in ALGS was associated with this effect on liver transplant/death
What is a 1.9 fold higher risk of liver transplant/death?
This genetic multisystem disorder causes a broad forehead, deep-set eyes, and a pointed chin, often alongside liver and heart abnormalities
What is ALGS?
The study completion rate for ASSERT
What is 100%?
This administration vehicle commonly used by pediatrics is clearly stated as not compatible with BYVLAY in the product monograph
What is bottle/sippy cup?
The dose of BYLVAY that was used in PEDFIC 2
What is 120 µg/kg per day?
In GALA, nearly three-quarters of liver transplants (247 out of 343) were performed by this age
What is 5 years old?
This early-onset genetic disorder is marked by severe pruritus and progressive cholestasis, with manifestations are largely confined to the liver
What is PFIC?
The study durations of ASSERT, a double-blind placebo-controlled trial, and ASSERT-EXT, an open-label extension
What is 24 weeks and 72 weeks?
This IBAT inhibitor is taken with food, while the other IBAT inhibitor should be separated from meals by 30 minutes
What is odevixibat with a meal and maralixibat 30 minutes before or after a meal?
The number of patients who discontinued BYLVAY in ASSERT due to intolerability
What is 0?
The median age, in years, at which children in the GALA study underwent liver transplantation
What is 2.8 years?
The gene mutation in PFIC associated with a poor response to IBAT inhibitors such as odevixibat or maralixibat
What is ABCB11, which encodes the bile salt export pump [BSEP]?
A unique aspect about the Phase 2 ICONIC trial in ALGS evaluating maralixibat in the treatment of ALGS
What is 18-week open-label treatment phase enriching for responders, followed by a 4-week randomized, double-blind, placebo-controlled drug-withdrawal phase?
The typical daily dosing frequency for odevixibat compared with maralixibat in PFIC
What is once daily for odevixibat and twice daily for maralixibat?
The number of patients in ASSERT who needed a temporary dose reduction from 120 µg/kg per day to 40 µg/kg per day
What is one patient?
In cholestatic pruritus, a serum bile acid level at or below this proposed threshold (in µmol/L) is associated with improved native liver survival
What is 102 µmol/L?
The proportion of parents who recognize pruritis as the primary symptom affecting their children diagnosed with ALGS
What is one-third?