공시 • Dec 10
Albireo Pharma, Inc. Submits for U.S. FDA and EMA Product Approval of Once-Daily Odevixibat for PFIC
Albireo Pharma, Inc. announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking approval of odevixibat for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC). Odevixibat is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) being developed to treat rare pediatric cholestatic liver diseases, including PFIC, biliary atresia and Alagille syndrome. The EMA has validated the odevixibat MAA on the accelerated assessment timeline, which begins the formal review process. With FDA and EMA regulatory submissions complete, odevixibat has the potential to become the first approved drug treatment for patients with PFIC in the U.S and Europe. Odevixibat has previously received Fast Track, Rare Pediatric Disease and Orphan Drug Designations in the U.S. In addition to PFIC, odevixibat has Orphan Drug Designations for the treatment of Alagille Syndrome, biliary atresia and primary biliary cholangitis. The EMA has granted odevixibat accelerated assessment, Orphan Designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. The EMA’s Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plans for PFIC and biliary atresia. With U.S. and EU regulatory submissions for odevixibat in PFIC completed, the Company anticipates potential regulatory approvals, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021. Phase 3 data was recently presented at the AASLD that showed a durable response to odevixibat in patients with PFIC. Full results from PEDFIC 1, the first and largest, global, Phase 3 study ever conducted in PFIC, confirm both U.S. and EU primary endpoints were met in the randomized, double-blind, placebo-controlled trial. Additionally, long-term data from PEDFIC 2, an open-label Phase 3 extension study, demonstrate continued and durable reductions in sBAs, improvements in pruritus assessments and encouraging markers of liver and growth function in patients treated up to 48 weeks. Across both studies, odevixibat was generally well tolerated, and treatment-emergent adverse events (TEAEs) were mostly mild or moderate. Collectively, these studies reaffirm odevixibat’s potential to be the first drug treatment approved for patients living with PFIC, a devastating disease which is currently treated with surgical options including liver transplantation. Odevixibat is also currently being evaluated in the ongoing PEDFIC 2 Phase 3 open-label trial in patients with PFIC, and the BOLD Phase 3 trial in patients with biliary atresia. The Phase 3 trial of odevixibat in Alagille syndrome will be the third pivotal trial of odevixibat. The Company also provides an Expanded Access Program for eligible patients with PFIC in the U.S., Canada, Australia and Europe. Progressive familial intrahepatic cholestasis (PFIC) is a rare disorder that causes progressive, life-threatening liver disease. Patients have impaired bile flow, or cholestasis, caused by genetic mutations. The resulting bile build-up in liver cells causes liver disease and symptoms. The most prominent and problematic ongoing manifestation of the disease is pruritus, or intense itching, which often results in a severely diminished quality of life. Other symptoms include jaundice, poor weight gain and slowed growth. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all people with PFIC require treatment before age 30. There are no drugs currently approved for PFIC, only surgical options that include partial external biliary diversion (PEBD) and liver transplantation. Biliary atresia is a rare pediatric liver disease with symptoms typically developing about two to eight weeks after birth. Damaged or absent bile ducts outside the liver result in bile and bile acids being trapped inside the liver, quickly resulting in cirrhosis, and even liver failure. Children have clay-colored or no color in their stools, jaundice among other things and a few patients are pruritic. Biliary atresia is the most common pediatric cholestatic liver disease and is the leading cause of liver transplants among children as there are no approved drug treatments. Alagille Syndrome (ALGS) is a rare multisystem genetic disorder that can affect the liver, heart, skeleton, eyes, central nervous system, kidneys, and facial features. Liver damage is caused by a paucity of bile ducts preventing bile flow from the liver to the small intestine. Approximately 95% of patients with ALGS present with chronic cholestasis, usually within the first three months of life, and up to 88% also present with severe, intractable pruritus. Currently, there are no approved drug treatments. Odevixibat is an investigational product candidate being developed to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. A potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), odevixibat acts locally in the small intestine. Odevixibat does not require refrigeration and can be taken as a capsule for older children, or opened and sprinkled onto food, which are factors of key importance for adherence in a pediatric patient population. Odevixibat is currently being evaluated in the ongoing PEDFIC 2 open-label trial (NCT03659916) and the BOLD Phase 3 trial in patients with biliary atresia (NCT04336722). Initiation of a pivotal Phase 3 trial of odevixibat for Alagille syndrome is also anticipated by the end of 2020.