According to a story from GlobeNewswire, the rare disease liver company Albireo Pharma, Inc., recently announced positive results from a phase 3 study testing the company’s experimental drug odevixibat as a treatment for progressive familial intrahepatic cholestasis (PFIC), a rare disease affecting the liver. The treatment was able to satisfy the primary and secondary endpoints in both the US and EU. The drug appeared to be effective in all three types of the disease.
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.
The phase 3 study included a total of 69 patients. The endpoints of this trial was reductions in pruritus (itching) and serum bile acids (sBAs) over a 24 week treatment period. Treatment with odevixibat was able to satisfy these endpoints, achieving positive pruritus assessments at a mean of 53.5 percent and change in serum bile acids of 33.3 percent. 93 percent of patients in the study are continuing treatment in an long term extension which has demonstrated that the drug has continued, sustained benefits.
Odevixibat is classified as a non-systemic inhibitor of ileal bile acid transport. It is orally available and is intended to be taken once per day. The experimental therapy is also being tested for other rare liver diseases that impact children, such as Alagille syndrome and biliary atresia. Odevixibat has earned Orphan Drug, Rare Pediatric Disease, and Fast Track designations in the US as well as Orphan designation and eligibility for the Priority Medicines scheme in the EU.