According to a story from Globe Newswire, the rare disease company Albireo Pharma, Inc. has recently announced the release of new data from its phase 3 trials. These trials were evaluating odevixibat (marketed as Bylvay) as a treatment for child patients living with the rare liver disorder progressive familial intrahepatic cholestasis (PFIC). The new data illustrated the benefits of dose escalation with the drug, which provided enhanced symptom relief and quality of life improvements with similar tolerability.
About Progressive Familial Intrahepatic Cholestasis
Progressive familial intrahepatic cholestasis (PFIC) are a group of hereditary diseases in which the flow of bile is affected. There are several different types of PFIC which are linked to different genetic mutations. These mutations generally cause a defect affecting biliary epithelial transporters. The symptoms of these diseases begin to appear in childhood, but some patients are not diagnosed until years later. These symptoms include lack of normal bile flow, cirrhosis, failure to thrive, jaundice, fat malabsorption, and severe itching. PFIC can eventually lead to complications such as osteopenia, a condition of lowered mineral density in bones. Treatment is generally supportive and symptomatic. Medications used to relieve symptoms include ursodeoxycholic acid, naloxone, and rifampin. Other approaches include certain surgical procedures and vitamin supplementation. If liver function begins to decline, a transplant may be necessary. To learn more about PFIC, click here.
New Data Revelations
Odevixibat is an inhibitor of ileal bile acid transport that is approved in both the EU and US as a treatment for progressive familial intrahepatic cholestasis. The PEDFIC 1 trial evaluated the treatment’s ability to provide symptoms relief and the PEDFIC 2 trial is a long-term extension study. The researchers found that in a pooled analysis, 21 of the patients that had responded to a 40 μg/kg/day dose either maintained the response or saw further improvements when the dose was increased to 120 μg/kg/day.
One of the most difficult symptoms of this disease is pruritus, or itching. This can involve almost the entire body and can be extremely severe. Needless to say, this can have a devastating impact on quality of life and can cause major sleep difficulty. The study found that with dose escalation, more patients found pruritus symptom relief at the increased dose.
Overall, these findings reinforce the effectiveness of the treatment and the benefit of an increased dose if a small dose doesn’t produce a response.
