Canbridge Pharmaceuticals Inc. obtained marketing approval from China’s NMPA for CAN-108 (Livmarli; maralixibat chloride oral solution) to treat cholestatic pruritus in 1-year-old or older patients with Alagille syndrome, a development that the company touted for how quickly it followed similar approvals elsewhere.
Canbridge Pharmaceuticals Inc. obtained marketing approval from China’s NMPA for CAN-108 (Livmarli; maralixibat chloride oral solution) to treat cholestatic pruritus in 1-year-old or older patients with Alagille syndrome, a development that the company touted for how quickly it followed similar approvals elsewhere.
Alagille syndrome (ALGS) is a rare JAG1 (encodes for a Notch ligand) autosomal dominant disease affecting approximately 1 in 30,000-40,000 individuals. ALGS developmental defects cause an absence of bile ducts (intrahepatic duct paucity, IHDP) with an inability to transport bile from the liver to bile ducts (cholestasis) as well as heart problems.
Mirum Pharmaceuticals Inc. plans to “move rapidly toward regulatory filings” to expand use of IBAT inhibitor Livmarli (maralixibat) into progressive familial intrahepatic cholestasis (PFIC), President and CEO Chris Peetz told investors during a conference call Oct. 24 to discuss the positive top-line results from the phase III March study, which not only hit its primary endpoint in patients with PFIC2 but showed statistical improvements in pruritis across a range of PFIC subtypes.
The next stop for Albireo Pharma Inc. is chats with the U.S. FDA and the EMA following positive phase III data for Bylvay (odevixibat) in treating the rare disease Alagille syndrome. Should Bylvay, a nonsystemic ileal bile acid transport inhibitor, be approved for the indication, it would be the second approval. It was greenlighted by the FDA in 2021 for treating pruritus in progressive familial intrahepatic cholestasis.
Although progressive familial intrahepatic cholestasis (PFIC) tends to draw more hoopla in the race, and Mirum Pharmaceuticals Inc. looks due to beat Albireo Pharma Inc. to market in Alagille syndrome (ALGS), the most revenue likely lies in a third rare pediatric liver disease where the firms compete: biliary atresia (BA). Mirum, of Foster City, Calif., completed its rolling NDA several weeks ago for maralixibat – an inhibitor of the apical sodium-dependent bile acid transporter designed to drive more excretion of bile acids as a way of lowering their level systemically – in cholestatic pruritus in patients with ALGS one year of age and older.
Fresh off hitting key regulatory milestones in Europe and the U.S. for its investigational cholestatic liver disease therapy, maralixibat, Mirum Pharmaceuticals Inc. has secured a $210 million deal with Oberland Capital Management LLC supporting the drug's potential commercialization and the company's pipeline development plans.
During its recent fourth-quarter and full-year earnings report, Foster City, Calif.-based Mirum Pharmaceuticals Inc. provided an update on the apical sodium dependent bile acid transporter (ASBT) inhibitor maralixibat for Alagille syndrome (ALGS), which will be the subject of a rolling NDA starting in the third quarter of 2020.
With two FDA meetings in its hip pocket to discuss maralixibat for treating pruritus associated with Alagille syndrome, Mirum Pharmaceuticals Inc. got a resounding boost from the market Monday as its stock (NASDAQ:MIRM) closed up 111%, at $17.28.