A vaccine for dengue fever, an advanced therapy for a complication after transplants, and a potential first-in-class drug for a form of psoriasis were among medicines recommended for approval by European regulators on Oct. 14.
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.
As expected, Mirum Pharmaceuticals Inc. won FDA clearance for Livmarli (maralixibat), a minimally absorbed ileal bile acid transporter inhibitor, for cholestatic pruritus in patients with the rare liver disease Alagille syndrome, 1 year of age and older.
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.
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.
