In the past, treatment for progressive familial intrahepatic cholestasis (PFIC) was symptomatic or surgical, meaning that there weren’t too many treatment options for this patient population. This all changed with the development of Bylvay (odevixibat).
This oral treatment offers a less invasive option, and now it is available to PFIC patients six months and older within Europe, following a recommendation from the National Institute for Health and Care Excellence (NICE).
About NICE’s Recommendation
NICE has officially recommended Bylvay for the treatment of all progressive forms of PFIC in patients six months and older. This suggestion falls under the Highly Specialised Technologies (HST) pathway, which means that the National Health Service (NHS) will fund the drug for use within 90 days.
This decision is aligned with other agencies, such as the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and the European Marketing Authorization (EMA). It is based off of two trials: the PEDFIC 1 and PEDFIC 2 studies.
These trials are the largest global, Phase 3 trials to be conducted within PFIC patients, and they produced the following data:
- Both primary endpoints (pruritus and serum bile acid) were met in the PEDFIC 1 trial
- The PEFFIC 2 trial, a long-term extension of the prior trial, saw sustained results in regard to the primary endpoints
- Improvements were seen in markers of liver function, growth, and sleep
- The treatment was well-tolerated across both trials
- There were no serious adverse events (AEs) related to treatment
- The most common AEs were diarrhea, abdominal pain, vomiting, fat-soluble vitamin deficiency, and liver test abnormalities
In the end, this recommendation is a very positive one for PFIC patients in Europe. It marks a new, non-surgical treatment option that will reduce the burdens this population carries.
About PFIC
PFIC is a group of conditions that impact the liver, characterized by defective biliary epithelial transporters. Children are most commonly impacted by this disease, and they see symptoms such as:
- Jaundice
- Failure to thrive
- The obstruction of bile to the liver (cholestasis)
- Pruritus
- Broken bones
- Issues with vision and balance
- Issues with depression have also been linked to PFIC
These symptoms are the result of one of numerous possible mutated genes (ATP8B1, ABCB11, and ABCB4), all of which are inherited in an autosomal recessive pattern. These alterations affect proteins needed for the formation and transportation of bile, making it so that the process does not happen as it should. Because of this blockages can form, and bile can buildup, which means that all of the toxic substances that are supposed to leave through the bile remain in the body.
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