The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted odevixibat (Bylvay®) marketing authorization to treat progressive familial intrahepatic cholestasis (PFIC), according to a press release from Albireo, the makers of the treatment.
This means the treatment is now approved across the US, EU, and UK for these patients. “The approval of Bylvay in the UK marks an important milestone to provide global access to the first approved drug for children with PFIC who desperately need it,” said Ron Cooper, president and chief executive officer of Albireo.
Odexibat has also been granted orphan drug designation for the treatment of Alagille syndrome (ALGS), biliary atresia, and primary biliary cholangitis in the US and EU.
Learn more about ALGS experimental therapies
The treatment is being investigated in a phase 3 trial in patients with ALGS, an open-label trial in patients with PFIC, and another phase 3 trial in patients with biliary atresia. The trials in patients with ALGS and biliary atresia are on track and topline data are expected in 2022 and 2024 respectively.
Odexibat is the first treatment approved by the US Food and Drug Administration (FDA) to treat pruritus in patients with all types of PFIC. It is a nonsystemic ileal bile acid transport inhibitor. Ileal bile acid transporter protein mediates the uptake of conjugated bile acids from the small intestine and transports them to the liver. By inhibiting bile acid transporter protein, it could prevent bile acid uptake, allowing more bile acids to be excreted thereby decreasing levels in the serum and ultimately reducing pruritus.
The approval of the treatment in patients with PFIC was based on the results of 2 phase 3 clinical trials that investigated its safety and efficacy in children with PFIC types 1 or 2.
“The approval of Bylvay gives parents tremendous hope as the first drug treatment specifically for PFIC to be deemed safe and effective,” added Alison Taylor, chief executive of the Children’s Liver Disease Foundation (CLDF).
Albireo receives UK MHRA approval of Bylvay® (odevixibat). News release. Albireo; September 8, 2021.