Odevixibat

Key takeaways

Odevixibat belongs to chemistry; place it in that map before memorising details.
Learn the definition first, then one example that makes the definition concrete.
Connect Odevixibat to a quantity you can measure, compute or draw — that is where exam questions come from.
Reproduce the core statement of Odevixibat from memory before moving on to harder problems.

Reference excerpt

Odevixibat, sold under the brand name Bylvay among others, is a medication for the treatment of progressive familial intrahepatic cholestasis. It is taken by mouth. Odevixibat is a reversible, potent, selective inhibitor of the ileal bile acid transporter (IBAT). It was developed by Albireo Pharma. The most common side effects include diarrhea, abdominal pain, hemorrhagic diarrhea, soft feces, and hepatomegaly (enlarged liver). Odevixibat was approved for medical use in the United States and in the European Union in July 2021. The U.S. Food and Drug Administration considers it to be a first-in-class medication.

Medical uses In the United States, odevixibat is indicated for the treatment of pruritus in people three months of age and older with progressive familial intrahepatic cholestasis. In the European Union it is indicated in people six months of age and older.

Mechanism of action Odevixibat is a reversible inhibitor of the ileal sodium/bile acid co-transporter. This transporter is responsible for reabsorption of the majority of bile acids in the distal ileum. The reduced absorption of the bile acids in the distal ileum compounds and leads to a decrease in stimulation of FXR (farnesoid X receptor), decreasing the inhibition of bile acid synthesis. Odevixibat works as a reversible, selective, small molecule inhibitor of the ileal bile acid transporter (IBAT).

Pharmacokinetics Odevixibat is > 99% protein-bound in vitro. A dose of odevixibat that is 7.2 mg reaches a cmax concentration of 0.47 ng/mL with an AUC (0-24h) of 2.19 h*ng/mL. Adult and pediatric patients given the therapeautic dose of odevixibat did not display plasma concentrations of the drug. Odevixibat is eliminated majorly unchanged. Odevixibat has an average half-life of 2.36 hours. The peak plasma time ranges from 1 to 5 hours after a single 7.2 mg dose in healthy adults. In healthy adults receiving a single 7.2 mg dose, the peak plasma concentration is 0.47 ng/mL, and the area under the concentration-time curve (AUC) is 2.19 ng·hr/mL. The plasma concentration of odevixibat in patients aged 6 months to 17 years ranges from 0.06 to 0.72 ng/mL. With once-daily dosing, there is no accumulation of odevixibat. Odevixibat is metabolized through a process called mono-hydroxylation.The drug is primarily eliminated through the feces (97% unchanged), with a minimal amount excreted in the urine (0.002%). Consuming a high-fat meal (800-1000 calories with approximately 50% of the total caloric content from fat) the peak plasma concentration is decreased by 72%, the AUC by 62%, and delays the peak plasma time by 3 to 4.5 hours. However, the impact of food on systemic exposures to odevixibat is not clinically significant.

Contraindications Odevixibat cannot be given to a child on a liquid diet.

Adverse effects Common side effects of odevixibat include diarrhea, stomach pain, vomiting, liver test abnormalities, abnormal liquid function tests, and a deficiency in vitamins A, D, E and K.

Pregnancy and lactation There are no enough human data on odevixibat use during pregnancy to build a drug-associated risk of major birth defects, miscarriage, or adverse developmental outcomes. There are no data on the presence of odevixibat in human milk, and how it affects milk production and breastfed babies.

History Preclinical studies and early clinical trials were conducted to evaluate the safety and efficacy of odevixibat, to establish the appropriate dosage, assess its mechanism of action, and evaluate its effects on bile acid levels and symptoms in people with progressive familial intrahepatic cholestasis. A 24-week clinical trial, played a role in demonstrating the effectiveness and safety of odevixibat in treating pruritus in children with progressive familial intrahepatic cholestasis. The US Food and Drug Administration (FDA) granted the application for odevixibat orphan drug designation. The FDA classified odevixibat as an orphan drug for the rare conditions of Alagille syndrome, biliary atresia, and primary biliary cholangitis. Odevixibat was granted its initial approval in July 2021, in the European Union for the treatment of progressive familial intrahepatic cholestasis in people aged six months and older. In July 2021, it received approval in the United States for the treatment of pruritus (itching) in people aged three months and older with progressive familial intrahepatic cholestasis.

Society and culture

Legal status In May 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended granting a marketing authorization in the European Union for odevixibat for the treatment of progressive familial intrahepatic cholestasis in people aged six months or older. It was authorized for medical use in the European Union in July 2021. In July 2024, the CHMP adopted a positive opinion, recommending the granting of a marketing authorization under exceptional circumstances for the medicinal product Kayfanda, intended for the treatment of cholestatic pruritus in people with Alagille syndrome aged six months or older. The applicant for this medicinal product is Ipsen Pharma. Kayfanda was authorized for medical use in the European Union in September 2024.

Research A phase III randomized control trial showed odevixibat reduced pruritis and serum bile acids in children with progressive familial intrahepatic cholestasis.

References

External links Clinical trial number NCT03566238 for "This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC 1 or 2 (PEDFIC 1)" at ClinicalTrials.gov

Illustrations

Worked examples

Example 1 — a first encounter with Odevixibat

Start with the simplest possible case. Write down what Odevixibat claims or describes in one sentence, then invent the smallest concrete situation in which that sentence is true. In chemistry, the smallest case is usually a single object, a single equation or a single measurement. Check that every symbol or term in your sentence has a meaning in that case.

Example 2 — changing one variable

Take the situation from Example 1 and change exactly one quantity: double it, halve it, or set it to zero. Predict what should happen to Odevixibat before you calculate. Comparing your prediction with the result is the fastest way to find out whether you understand the idea or only the words.

Example 3 — an exam-style question

Typical questions about Odevixibat ask you to (a) state it precisely, (b) apply it to given data, and (c) explain a limitation. Practise writing all three answers in under five minutes; the third part is what separates a full-mark answer from an average one.

Applications of Odevixibat

In research: Odevixibat appears in chemistry research whenever the underlying quantities have to be modelled precisely. Papers usually cite it as a starting assumption and then explore where it breaks down.
In technology and industry: Engineering practice reuses Odevixibat in design rules, simulations and safety margins. Knowing the idea lets you read a specification sheet and understand why the numbers look the way they do.
In the classroom: Odevixibat is common in secondary-school and first-year university syllabi. It links to neighbouring topics 4-Hydroxyphenyl compounds, Amides, Carboxylic acids, so understanding it makes those chapters shorter.
In everyday life: Look for Odevixibat outside the textbook — in sport, cooking, traffic, electronics or the sky above you. An example you found yourself is remembered far longer than one you were given.

How to study Odevixibat in 20 minutes

Read the reference excerpt below once, without taking notes.
Close the page and write down what Odevixibat means in your own words.
Compare your version with the excerpt and mark what you missed.
Work through the three examples above with pen and paper.
Explain Odevixibat out loud to somebody else — or to Teacher Smith in the lgStudy chat.

Frequently asked questions

What is Odevixibat in simple terms?

Odevixibat, sold under the brand name Bylvay among others, is a medication for the treatment of progressive familial intrahepatic cholestasis. It is taken by mouth.

Why does Odevixibat matter?

Because it connects several chemistry ideas at once: it gives you a definition you can apply, a quantity you can calculate, and a way to check whether a result is plausible.

How should I study Odevixibat?

Read the excerpt, restate it from memory, then work through the examples and applications listed on this page. The five-step study plan above takes about twenty minutes.

What does this page cover?

It gives you a compact reference excerpt plus original lgStudy explanations, examples, applications and study material on Odevixibat.