In a video posted on Healio, Dr. Lorenzo D’Antiga from the Pappa Giovanni XXIII Hospital talked about the findings from a recent clinical study in which the drug odevixibat (also called Bylvay) was able to produce significant improvements in people living with the rare disease progressive familial intrahepatic cholestasis. This therapy could help improve outcomes for these patients, who don’t have access to disease-modifying treatments that can slow or halt disease progression.
About Progressive Familial Intrahepatic Cholestasis (PFIC)
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.
About The Clinical Trial
Odevixibat is intended to be used once per day and is classified as a non-systemic inhibitor of ileal bile acid transport. In the trial, the therapy was being evaluated principally to investigate its effects on serum bile acid and pruritus (severe itching). A total of 77 patients participated in the trial. These patients were treated for a period of 37 weeks and most had the type 2 form of PFIC. These patients had all been previously treated with other medicines.
At 48 weeks the treatment triggered a substantial reduction in serum bile acid; meanwhile, the placebo group actually saw their serum bile acid increase during this time. The drug also reduced pruritus, which can be a severe and debilitating symptom that can have devastating effects on quality of life and sleep. As this symptom improved, the patients saw their quality and duration of sleep go up as well. The safety profile of the drug was encouraging as well.
Ultimately, this treatment could help make PFIC a much more manageable condition and could prevent the development of end-stage liver disease in these patients.