KAYFANDA: Uses, Dosage & Side Effects
An ileal bile acid transporter (IBAT) inhibitor for the treatment of cholestatic pruritus in patients aged 6 months and older with Alagille syndrome
KAYFANDA (odevixibat) is a prescription medication approved in the European Union for the treatment of cholestatic pruritus in patients aged 6 months and older with Alagille syndrome (ALGS). It contains the active substance odevixibat, a potent and selective inhibitor of the ileal bile acid transporter (IBAT). By blocking bile acid reabsorption in the intestine, KAYFANDA lowers serum bile acid levels and relieves the severe, debilitating itching that profoundly impairs quality of life in patients with this rare genetic liver disease. The medication acts locally in the gut with minimal systemic absorption. KAYFANDA was approved in September 2024 under exceptional circumstances due to the extreme rarity of Alagille syndrome, and the medicine is under additional monitoring.
Quick Facts: KAYFANDA
Key Takeaways
- KAYFANDA (odevixibat) is the first and only medication specifically approved in the EU for the treatment of cholestatic pruritus in Alagille syndrome, a rare genetic disorder affecting approximately 1 in 30,000 to 1 in 50,000 live births.
- It works locally in the gut by blocking the ileal bile acid transporter (IBAT), reducing serum bile acid levels without significant systemic absorption, which contributes to its favorable safety profile.
- In the pivotal ASSERT Phase III trial, KAYFANDA demonstrated statistically significant improvements in pruritus severity and reductions in serum bile acid levels compared with placebo in patients with Alagille syndrome.
- Available in multiple capsule strengths (200, 400, 600, and 1200 micrograms) to support individualized weight-based dosing from 6 months of age, with capsule contents that can be mixed with soft food for young children.
- Regular monitoring of liver function tests and fat-soluble vitamin levels (A, D, E, K) is required during treatment, as the medication is approved under exceptional circumstances and is under additional monitoring by the EMA.
What Is KAYFANDA and What Is It Used For?
KAYFANDA contains the active substance odevixibat (as odevixibat sesquihydrate), a potent, selective, and reversible inhibitor of the ileal bile acid transporter (IBAT), also known as the apical sodium-dependent bile acid transporter (ASBT). IBAT is a protein located in the epithelial cells of the terminal ileum (the last part of the small intestine) that is responsible for the active reabsorption of bile acids from the intestinal lumen back into the portal circulation. This reabsorption process is a critical component of the enterohepatic circulation of bile acids, the cycle through which bile acids are secreted by the liver, stored in the gallbladder, released into the intestine to aid fat digestion, and then reabsorbed to be recycled by the liver.
In healthy individuals, approximately 95% of bile acids secreted into the intestine are reabsorbed by IBAT and returned to the liver via the portal vein. However, in patients with Alagille syndrome, a rare autosomal dominant genetic disorder caused primarily by mutations in the JAG1 gene (and less commonly in the NOTCH2 gene), the liver and bile ducts do not develop normally. This leads to a paucity (reduced number) of intrahepatic bile ducts, resulting in impaired bile flow (cholestasis) and accumulation of bile acids in the liver and blood. The elevated serum bile acid concentrations cause intense, debilitating pruritus (itching) that is often the most distressing symptom experienced by patients and their families. The pruritus in Alagille syndrome can be so severe that it disrupts sleep, impairs daily functioning, affects growth and development in children, and significantly reduces quality of life for both patients and caregivers.
By blocking IBAT in the terminal ileum, odevixibat prevents the reabsorption of bile acids from the intestine. Instead of being recycled back to the liver, bile acids are diverted to the colon and excreted in the feces. This interruption of the enterohepatic circulation leads to a net reduction in the total body bile acid pool and a decrease in serum bile acid concentrations. The resulting lowering of circulating bile acids is the primary mechanism by which KAYFANDA alleviates cholestatic pruritus. Importantly, odevixibat acts locally in the gastrointestinal tract with minimal systemic absorption. After oral administration, plasma concentrations of odevixibat are generally very low or below the limit of quantification, confirming its predominantly local mechanism of action. This non-systemic profile is a significant advantage in terms of systemic side effects.
Alagille syndrome is estimated to affect approximately 1 in 30,000 to 1 in 50,000 live births worldwide. It is a multisystem disorder that can affect the liver, heart, skeleton, eyes, kidneys, and facial features. The hepatic manifestations, including cholestasis and pruritus, are among the most clinically significant features and are present in over 90% of affected individuals. Before the approval of KAYFANDA, treatment options for cholestatic pruritus in Alagille syndrome were extremely limited and often inadequate. Off-label therapies such as ursodeoxycholic acid (UDCA), rifampicin, cholestyramine, and naltrexone provided variable and frequently insufficient relief. Many patients ultimately required surgical intervention, including partial external biliary diversion or liver transplantation, to manage intractable pruritus.
The approval of KAYFANDA was based on the results of the ASSERT trial, a pivotal Phase III, randomized, double-blind, placebo-controlled study conducted in patients with Alagille syndrome. The ASSERT trial was the first and only completed Phase III clinical trial in this patient population. In this trial, patients aged 6 months and older with a confirmed diagnosis of Alagille syndrome and cholestatic pruritus were randomized to receive either odevixibat 120 micrograms/kg/day or placebo for 24 weeks. The primary endpoint was the proportion of patients achieving a clinically meaningful reduction in pruritus severity, as measured by validated observer-reported outcome instruments (the Observer Scratch Scale and the Itch Reported Outcome). Secondary endpoints included changes in serum bile acid levels and other measures of cholestasis.
The ASSERT trial demonstrated that a significantly greater proportion of patients treated with KAYFANDA achieved a positive pruritus response compared with placebo. Patients in the KAYFANDA group also showed statistically significant reductions in serum bile acid concentrations. These findings provided the evidence base for the European Commission to grant marketing authorization for KAYFANDA in September 2024 under exceptional circumstances, recognizing that comprehensive data could not be obtained under normal conditions due to the extreme rarity of Alagille syndrome. KAYFANDA is designated as an orphan medicinal product and is under additional monitoring, indicated by the inverted black triangle symbol, meaning that new safety information is actively collected and reviewed.
Alagille syndrome is a rare genetic disorder primarily caused by mutations in the JAG1 gene. It affects multiple organ systems but is most commonly recognized by liver disease (bile duct paucity leading to cholestasis), cardiac defects (most often peripheral pulmonary stenosis), skeletal abnormalities (butterfly vertebrae), ocular findings (posterior embryotoxon), and characteristic facial features. The cholestatic pruritus is often the most debilitating symptom, significantly impacting quality of life. KAYFANDA represents the first specifically approved treatment targeting this symptom in Alagille syndrome.
What Should You Know Before Taking KAYFANDA?
Contraindications
KAYFANDA is contraindicated in patients with known hypersensitivity to odevixibat or to any of the excipients listed in the product composition. If you have experienced an allergic reaction to any component of KAYFANDA, you must not use this medication. Additionally, KAYFANDA should not be used in patients with complete biliary obstruction, as the mechanism of action requires at least some bile acid flow into the intestine for the drug to exert its therapeutic effect. In patients with complete obstruction of bile flow, the medication cannot reduce serum bile acids through its intended mechanism.
It is important to note that KAYFANDA contains the same active substance (odevixibat) as Bylvay, which is approved for a different indication (progressive familial intrahepatic cholestasis). Patients should not use both products simultaneously, as this would result in an unintended double dose of odevixibat.
Warnings and Precautions
KAYFANDA may cause elevations in liver enzymes (ALT, AST). Liver function tests should be performed before starting treatment and monitored regularly during therapy. If liver enzyme elevations become clinically significant or are accompanied by signs of liver injury, dose reduction or treatment discontinuation should be considered. It may be difficult to distinguish drug-related liver enzyme changes from the underlying liver disease in Alagille syndrome.
Before and during treatment with KAYFANDA, your healthcare provider should be aware of the following important considerations:
- Liver enzyme monitoring: In clinical trials, elevations in alanine aminotransferase (ALT) greater than 3 times the upper limit of normal were observed in approximately 8–11% of patients treated with odevixibat. However, it is clinically challenging to differentiate these elevations from the natural fluctuations of liver enzymes that occur as part of the underlying Alagille syndrome. Liver function tests (including ALT, AST, bilirubin, and GGT) should be checked at baseline and at regular intervals during treatment, as recommended by your doctor.
- Fat-soluble vitamin deficiency: By increasing fecal bile acid excretion, KAYFANDA may also reduce the absorption of fat-soluble vitamins (A, D, E, and K) from the gastrointestinal tract. Bile acids play an essential role in the emulsification and absorption of dietary fats and fat-soluble vitamins. Patients with Alagille syndrome are already at increased risk of fat-soluble vitamin deficiency due to their underlying cholestatic liver disease. Vitamin levels should be monitored before starting treatment and at regular intervals throughout therapy. Supplementation with fat-soluble vitamins may be necessary and should be guided by laboratory monitoring.
- Diarrhoea: Diarrhoea is the most common side effect of KAYFANDA. The increased excretion of bile acids into the colon can cause osmotic diarrhoea. In most cases, diarrhoea is mild to moderate and self-limiting. However, if severe or persistent diarrhoea occurs, it may lead to dehydration, particularly in young children. Adequate fluid intake should be ensured, and dose adjustment or temporary treatment interruption may be considered if diarrhoea is severe.
- Additional monitoring: KAYFANDA is under additional monitoring due to its approval under exceptional circumstances. The European Medicines Agency reviews the marketing authorization annually. Any suspected adverse reactions should be reported to facilitate ongoing safety assessment.
Pregnancy and Breastfeeding
There are limited data on the use of KAYFANDA in pregnant women. Animal studies have not indicated direct or indirect harmful effects with respect to reproductive toxicity. However, as a precautionary measure, KAYFANDA should preferably be avoided during pregnancy unless clearly necessary. Women of childbearing potential should discuss contraception and family planning with their healthcare provider before starting treatment.
It is not known whether odevixibat or its metabolites are excreted in human breast milk. Given the minimal systemic absorption of odevixibat after oral administration, exposure of the breastfed infant is expected to be negligible. However, a risk to the nursing infant cannot be completely excluded. The decision to continue or discontinue breastfeeding or to continue or discontinue KAYFANDA therapy should be made in consultation with your healthcare provider, taking into account the benefit of breastfeeding for the child and the benefit of therapy for the mother.
Children
KAYFANDA is specifically indicated for patients aged 6 months and older with Alagille syndrome. The safety and efficacy of KAYFANDA in patients younger than 6 months have not been established, and no clinical data are available for this age group. Dosing in pediatric patients is based on body weight (40 micrograms per kilogram per day), and the availability of multiple capsule strengths (200, 400, 600, and 1200 micrograms) facilitates individualized dosing across a wide range of body weights. For young children who cannot swallow capsules whole, the capsule contents can be sprinkled onto a small amount of soft food.
Driving and Operating Machinery
KAYFANDA has no or negligible influence on the ability to drive and use machines. Based on its pharmacological properties and the known side effect profile, odevixibat is not expected to impair driving ability or the operation of machinery. The medication acts locally in the gut with minimal systemic exposure.
How Does KAYFANDA Interact with Other Drugs?
Because odevixibat acts locally in the gastrointestinal tract with minimal systemic absorption, the potential for traditional systemic pharmacokinetic drug interactions is low. Plasma concentrations of odevixibat after oral administration are generally very low or undetectable, meaning that CYP450-mediated drug interactions are unlikely. However, there are important pharmacodynamic and absorption-related considerations that patients and healthcare providers should be aware of.
The primary interaction concern with KAYFANDA relates to its mechanism of action: by inhibiting bile acid reabsorption, KAYFANDA alters the bile acid composition in the intestinal lumen. This can potentially affect the absorption or efficacy of other medications that interact with bile acids or require bile acids for their own absorption. Below is a summary of relevant interaction considerations:
| Drug / Drug Class | Type of Interaction | Clinical Recommendation |
|---|---|---|
| Bile acid sequestrants (cholestyramine, colesevelam, colestipol) | Pharmacodynamic: may bind odevixibat in the gut, reducing its efficacy | Administer KAYFANDA at least 1 hour before or 3 hours after bile acid sequestrants |
| Ursodeoxycholic acid (UDCA) | Pharmacodynamic: both affect bile acid metabolism; combined use may have additive effects | May be used concomitantly with appropriate monitoring; discuss with your doctor |
| Fat-soluble vitamins (A, D, E, K) | Absorption: reduced bile acids in gut may impair vitamin absorption | Monitor vitamin levels and supplement as needed; consider timing of supplementation |
| Oral contraceptives | Potential absorption effect: severe diarrhoea may reduce absorption | Additional contraceptive measures may be needed if persistent diarrhoea occurs |
| Ciclosporin, tacrolimus (immunosuppressants) | Theoretical: changes in bile acid pool may affect absorption of highly lipophilic drugs | Monitor drug levels if co-administered; no formal interaction study conducted |
| Midazolam (CYP3A4 substrate) | No significant interaction identified in a dedicated clinical study | No dose adjustment needed |
In a dedicated drug interaction study, co-administration of odevixibat with midazolam (a sensitive CYP3A4 substrate) did not result in clinically significant changes in midazolam exposure, confirming the low potential for systemic drug-drug interactions. However, formal interaction studies with most of the medications listed above have not been performed, and recommendations are based on the pharmacological properties of odevixibat and theoretical considerations.
Patients with Alagille syndrome who are receiving KAYFANDA may also be taking other medications for their hepatic or extrahepatic manifestations, including cardiac medications, growth hormone therapy, or medications for renal complications. While no specific interactions with these medications have been identified, it is essential to inform your doctor about all medications, supplements, and herbal products you are taking. This enables comprehensive monitoring and ensures optimal treatment outcomes.
To minimize potential interactions, KAYFANDA should be taken with a meal (to enhance its action in the presence of bile acids). If you are also taking bile acid sequestrants (such as cholestyramine), separate the doses: take KAYFANDA at least 1 hour before or 3 hours after the bile acid sequestrant. For other oral medications, maintain a consistent dosing schedule and discuss any concerns about timing with your healthcare provider.
What Is the Correct Dosage of KAYFANDA?
KAYFANDA should always be used exactly as your doctor has instructed. The dosage is individualized based on body weight and clinical response. Treatment should be initiated and supervised by a physician experienced in the management of cholestatic liver diseases. The medication is taken orally, once daily, with a meal to optimize its interaction with bile acids in the intestinal lumen.
Adults and Adolescents
While Alagille syndrome is predominantly diagnosed in childhood, some patients continue treatment into adolescence and adulthood. The dosing principles remain the same across age groups, based on body weight:
| Phase | Dose | Frequency | Duration |
|---|---|---|---|
| Starting dose | 40 mcg/kg/day | Once daily with a meal | At least 3 months |
| Dose escalation (if needed) | 120 mcg/kg/day | Once daily with a meal | Ongoing (reassess periodically) |
| Maximum recommended dose | 120 mcg/kg/day (max 7,200 mcg/day) | Once daily with a meal | Ongoing |
The starting dose is 40 micrograms per kilogram of body weight per day. If, after at least 3 months of treatment at the starting dose, the clinical response is considered insufficient (i.e., pruritus has not improved sufficiently), the dose may be increased to 120 micrograms/kg/day. The maximum recommended daily dose is 120 micrograms per kilogram of body weight, not exceeding 7,200 micrograms (7.2 mg) per day.
Children (6 Months and Older)
KAYFANDA is specifically designed for use in pediatric patients, as Alagille syndrome is typically diagnosed in infancy or early childhood. The weight-based dosing approach ensures appropriate dosing across the wide range of body weights encountered in pediatric practice. KAYFANDA capsules are available in four strengths to facilitate accurate dosing:
| Capsule Strength | Capsule Identification | Typical Use |
|---|---|---|
| 200 micrograms | Marked hard capsule | Infants and small children |
| 400 micrograms | Marked hard capsule | Young children |
| 600 micrograms | Marked hard capsule | Older children |
| 1200 micrograms | Marked hard capsule | Adolescents and adults |
For patients who cannot swallow capsules whole (particularly infants and young children), the capsule may be opened and its contents sprinkled onto a small amount of soft food (such as applesauce, yoghurt, or fruit puree). The food with the sprinkled capsule contents should be consumed immediately and not stored for later use. It is important not to crush or chew the capsule contents. The medication should be followed with a drink of water or other liquid to ensure complete intake.
Elderly Patients
There is no specific experience with KAYFANDA in elderly patients. Alagille syndrome is a congenital condition, and most patients receiving KAYFANDA are children and young adults. No specific dose adjustment is expected to be necessary in older patients, as the medication acts locally with minimal systemic absorption.
Missed Dose
If a dose of KAYFANDA is missed, it should be taken as soon as remembered on the same day with food. If the entire day has passed, the missed dose should be skipped and the next dose taken at the usual time the following day. Do not take a double dose to make up for a forgotten dose. Maintaining a consistent daily dosing schedule is important for optimal therapeutic effect. Setting a daily reminder or taking KAYFANDA with the same meal each day can help prevent missed doses.
Overdose
There is limited experience with overdose of KAYFANDA. In the event of an overdose, general supportive measures should be applied, including monitoring for signs and symptoms of adverse effects, particularly diarrhoea and dehydration. Given the primarily local mechanism of action and minimal systemic absorption, severe systemic toxicity from an overdose is considered unlikely. However, patients should seek medical advice promptly if an overdose is suspected. There is no specific antidote for odevixibat.
What Are the Side Effects of KAYFANDA?
Like all medicines, KAYFANDA can cause side effects, although not everyone who takes it will experience them. The side effects of KAYFANDA are primarily related to its mechanism of action: by increasing bile acid excretion in the feces, the medication can cause gastrointestinal symptoms and may affect fat-soluble vitamin absorption. The safety profile of KAYFANDA has been characterized in the ASSERT Phase III clinical trial and through ongoing post-marketing surveillance.
In the ASSERT trial, the overall incidence of adverse events was generally similar between the KAYFANDA and placebo groups, with the exception of diarrhoea, which was more common in patients receiving KAYFANDA. Drug-related diarrhoea was reported in approximately 11.4% of patients receiving odevixibat compared with 5.9% of patients receiving placebo. The overall safety profile observed in the trial was consistent with the known pharmacological effects of IBAT inhibition.
Very Common
May affect more than 1 in 10 people
- Diarrhoea
Common
May affect up to 1 in 10 people
- Abdominal pain
- Soft feces (softer stools than usual)
- Hepatomegaly (enlarged liver)
- Elevated liver enzymes (ALT increased)
Uncommon
May affect up to 1 in 100 people
- Nausea
- Flatulence (excessive gas)
- Abdominal distension (bloating)
- Fat-soluble vitamin deficiency (vitamins A, D, E, or K)
Rare
May affect up to 1 in 1,000 people
- Severe hepatic events (liver enzyme elevations requiring treatment modification)
- Dehydration (secondary to severe diarrhoea, particularly in young children)
Diarrhoea is the most frequently reported side effect and is a predictable pharmacological consequence of IBAT inhibition. When bile acids that would normally be reabsorbed in the ileum instead pass into the colon, they stimulate colonic fluid secretion and motility, resulting in looser and more frequent stools. In most patients, diarrhoea is mild to moderate in severity and tends to improve over time as the body adjusts to the medication. However, in some patients, particularly young children, persistent diarrhoea may lead to dehydration and electrolyte imbalances. Parents and caregivers should ensure adequate fluid intake and contact their healthcare provider if diarrhoea is severe, persistent, or accompanied by signs of dehydration (reduced urine output, dry mouth, lethargy).
Elevated liver enzymes (particularly ALT) were observed in approximately 8–11% of patients receiving odevixibat in clinical trials. These elevations are challenging to interpret in the context of Alagille syndrome, where fluctuations in liver enzymes are common as part of the underlying disease. Nevertheless, regular monitoring of liver function is recommended, and dose adjustment or treatment discontinuation should be considered if elevations are clinically significant or accompanied by symptoms of liver injury (jaundice, dark urine, persistent nausea, right upper abdominal pain).
Fat-soluble vitamin deficiency is an important potential consequence of long-term IBAT inhibitor therapy. By reducing bile acid availability in the intestinal lumen, KAYFANDA may impair the emulsification and absorption of dietary fats and the fat-soluble vitamins A, D, E, and K. Patients with Alagille syndrome are already predisposed to fat-soluble vitamin deficiency due to their underlying cholestatic liver disease. Regular monitoring of vitamin levels and appropriate supplementation are essential components of care for patients receiving KAYFANDA. Vitamin D deficiency may contribute to metabolic bone disease, vitamin A deficiency may affect vision and immune function, vitamin E deficiency may cause neurological symptoms, and vitamin K deficiency may impair blood clotting.
Contact your doctor immediately if you experience severe or persistent diarrhoea, signs of dehydration (especially in young children), yellowing of the skin or eyes (jaundice), dark urine, severe abdominal pain, or unusual bleeding or bruising (which may indicate vitamin K deficiency). Report any new or unexpected symptoms to your healthcare provider, as KAYFANDA is under additional monitoring.
How Should You Store KAYFANDA?
Proper storage of KAYFANDA ensures the medication maintains its quality, safety, and therapeutic effectiveness throughout its shelf life. Unlike some biologicals that require refrigeration, KAYFANDA capsules are stable at room temperature, making storage convenient for patients and families.
Follow these storage guidelines:
- Temperature: Store below 30 °C (86 °F). Do not expose to excessive heat. Avoid leaving the medication in a car during hot weather or in direct sunlight.
- Moisture protection: Keep the capsules in the original blister packaging until ready to take a dose. The packaging is designed to protect the capsules from moisture, which can affect the stability of the medication.
- Keep out of reach of children: As with all medications, store KAYFANDA in a secure location where children cannot access it.
- Expiration date: Do not use KAYFANDA after the expiration date (EXP) printed on the packaging. The expiration date refers to the last day of that month.
- Do not transfer: Do not transfer capsules to another container. The original packaging provides optimal protection and includes important identification information.
- Disposal: Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. These measures help protect the environment.
When traveling with KAYFANDA, keep the medication in its original packaging and carry it in your hand luggage rather than checked baggage to avoid exposure to extreme temperatures. If you are traveling to a hot climate, take precautions to keep the medication below 30 °C. Airport security screening (x-ray) is not expected to affect the medication.
If a capsule has been opened and sprinkled onto food, the mixture should be consumed immediately and not stored for later use. Any remaining food with sprinkled capsule contents that is not consumed within 30 minutes should be discarded.
What Does KAYFANDA Contain?
Understanding the complete composition of your medication is important, particularly for patients with known allergies or dietary restrictions. Below is a detailed breakdown of what KAYFANDA capsules contain.
Active Ingredient
The active substance is odevixibat (present as odevixibat sesquihydrate). Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT). It has a molecular weight of approximately 448.5 g/mol for the free base form. Each capsule contains one of the following strengths: 200 micrograms, 400 micrograms, 600 micrograms, or 1200 micrograms of odevixibat.
Inactive Ingredients (Excipients)
| Ingredient | Role | Notes |
|---|---|---|
| Odevixibat sesquihydrate | Active substance (IBAT inhibitor) | 200, 400, 600, or 1200 mcg per capsule |
| Microcrystalline cellulose | Filler / diluent | Provides bulk and aids capsule filling |
| Hypromellose (HPMC) | Capsule shell material | Plant-derived capsule shell |
| Titanium dioxide (E171) | Capsule shell colorant | White opacifier |
| Iron oxide (E172) | Capsule shell colorant | For strength identification |
| Printing ink | Identification marking | Contains shellac, iron oxide black |
Appearance and Pack Sizes
KAYFANDA capsules are hard capsules of different colors depending on the strength, to help distinguish between doses. They are supplied in blister packs. Not all pack sizes may be marketed in every country. Your pharmacist will provide the appropriate strength and pack size as prescribed by your doctor.
Marketing Authorization Holder and Manufacturer
KAYFANDA is manufactured and marketed by Ipsen Pharma, with headquarters at 65 quai Georges Gorse, 92100 Boulogne-Billancourt, France. The marketing authorization was granted by the European Commission in September 2024. Ipsen is a global specialty-driven biopharmaceutical company focused on oncology, rare diseases, and neuroscience. The same active substance (odevixibat) is also marketed by Ipsen under the brand name Bylvay for a different indication (progressive familial intrahepatic cholestasis).
Frequently Asked Questions About KAYFANDA
KAYFANDA (odevixibat) is used for the treatment of cholestatic pruritus (severe itching caused by bile acid buildup) in patients aged 6 months and older with Alagille syndrome. Alagille syndrome is a rare inherited liver disease where bile cannot drain properly from the liver, leading to high levels of bile acids in the blood that cause intense itching. KAYFANDA works by blocking the reabsorption of bile acids in the intestine, allowing them to be excreted in the stool, thereby lowering blood bile acid levels and relieving itching.
Both KAYFANDA and Bylvay contain the same active substance, odevixibat, and are manufactured by Ipsen. The key difference is their approved indications: KAYFANDA is specifically approved for cholestatic pruritus in Alagille syndrome (ALGS), while Bylvay is approved for progressive familial intrahepatic cholestasis (PFIC). Although the active ingredient is identical, the clinical evidence, dosing recommendations, and regulatory approvals are specific to each product and its respective indication. Patients should not switch between the two products without medical supervision.
In the ASSERT clinical trial, improvements in pruritus were observed over the 24-week treatment period. Some patients may notice improvement within the first few weeks of treatment, while for others it may take longer. Your doctor will typically evaluate the response after at least 3 months of treatment at the starting dose. If the response is insufficient, the dose may be increased. It is important to take KAYFANDA consistently every day as prescribed to achieve the best results.
Yes, KAYFANDA capsules are designed to accommodate young children who cannot swallow capsules whole. The capsule can be opened and its contents sprinkled onto a small amount of soft food such as applesauce, yoghurt, or fruit puree. The food should be consumed immediately after mixing and followed with a drink of water or other liquid. Do not crush or chew the capsule contents. Any remaining food-medication mixture not consumed within 30 minutes should be discarded. Your doctor or pharmacist can demonstrate the proper technique.
Regular monitoring is an essential part of KAYFANDA treatment. Your doctor will check liver function tests (ALT, AST, bilirubin, GGT) before starting treatment and at regular intervals during therapy. Fat-soluble vitamin levels (vitamins A, D, E, and K) should also be monitored and supplemented as needed. Serum bile acid levels may be measured to assess treatment response. Growth and nutritional status should be monitored in pediatric patients. Additionally, because KAYFANDA is approved under exceptional circumstances and is under additional monitoring, reporting any suspected adverse reactions helps ensure ongoing safety assessment.
No, KAYFANDA does not cure Alagille syndrome. It is a symptomatic treatment that specifically targets cholestatic pruritus (itching) by reducing serum bile acid levels. It does not address the underlying genetic cause of the disease or reverse the structural abnormalities in the bile ducts. Patients with Alagille syndrome will continue to need comprehensive medical management for their condition. However, by effectively relieving pruritus, KAYFANDA can significantly improve quality of life for patients and their families.
References
- European Medicines Agency (EMA). KAYFANDA (odevixibat) – Summary of Product Characteristics. Approved September 2024. Available at: EMA KAYFANDA EPAR.
- Ipsen Pharma. KAYFANDA (odevixibat) – Product Information. European Commission Marketing Authorization, September 2024.
- Gonzales E, Hardikar W, Engelen R, et al. Efficacy and Safety of Odevixibat in Alagille Syndrome (ASSERT): A Randomized, Double-blind, Placebo-controlled, Phase 3 Trial. Hepatology. 2024.
- Spinner NB, Gilbert MA, Loomes KM, Krantz ID. Alagille Syndrome. In: Adam MP, et al., editors. GeneReviews. University of Washington, Seattle; 1993–2024. Available at: NCBI GeneReviews.
- European Association for the Study of the Liver (EASL). EASL Clinical Practice Guidelines on Sclerosing Cholangitis. J Hepatol. 2022;77(3):761–806.
- Kamath BM, Baker A, Houwen R, Todorova L, Mattsson JP. Systematic Review: The Epidemiology, Natural History, and Burden of Alagille Syndrome. J Pediatr Gastroenterol Nutr. 2018;67(2):148–156.
- Karpen SJ, Kelly D, Mack C, Stein P. Ileal Bile Acid Transporter Inhibition as an Anticholestatic Therapeutic Target in Biliary Atresia and Other Cholestatic Disorders. Hepatol Int. 2020;14(5):677–689.
- World Health Organization (WHO). WHO Model List of Essential Medicines for Children. 2023. Available at: WHO EMLc.
- British National Formulary for Children (BNFc). Odevixibat. National Institute for Health and Care Excellence (NICE). 2025.
- U.S. National Library of Medicine. Odevixibat. MedlinePlus Drug Information. 2024. Available at: MedlinePlus.
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