Cholemic nephropathy (CN) is an acute renal dysfunction that occurs after high bile acid (BA) levels provoke epithelial cell damage and tubular obstruction in the kidneys.
Parties to Astrazeneca plc’s potential $1.8 billion takeover of Cincor Pharma Inc. are keeping mum about contingent value rights included in the deal for baxdrostat, the phase III-bound aldosterone synthase targeter in the works to treat hypertension and chronic kidney disease. Another acquisition that captured headlines as the J.P. Morgan Healthcare Conference launched in San Francisco: the $1.4 billion-plus agreement whereby Chiesi Farmaceutici S.p.A. will take ownership of Amryt Pharma plc. And there was more, as Ipsen SA pledged $952 million to make Albireo Pharma Inc. its own.
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.
Albireo Pharma Inc.’s Bylvay (odevixibat) becomes the first therapy to hit the commercial market for rare genetic liver disease progressive familial intrahepatic cholestasis (PFIC). A day after the European Commission approved the drug, the FDA gave the nod to the ileal bile acid transport inhibitor on its July 20 PDUFA date following a priority review.
DUBLIN – Skysona (elivaldogene autotemcel, Lenti-D), Bluebird Bio Inc.’s gene therapy for cerebral adrenoleukodystrophy, received a nod from the EMA’s Committee for Human Medicinal Products (CHMP) during its May meeting this week, paving the way for a formal European authorization in the coming weeks. It will constitute the first approval for the product. An FDA approval is some way behind – the company will not complete its BLA filing with the FDA until around midyear.
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.
Good news from a pivotal phase III trial of Albireo Pharma Inc.'s odevixibat has moved the world one step closer to going from no treatments for the rare genetic liver disease PFIC to possibly having two: Albireo's entrant and another candidate, maralixibat from Mirum Pharmaceuticals Inc.
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.
