Though it’s been used off-label for more than three decades to treat cerebrotendinous xanthomatosis, Mirum Pharmaceuticals Inc.’s chenodiol gained an official U.S. FDA nod Feb. 21 as the first drug approved specifically for treating the rare autosomal recessive lipid storage disease.
Label comparisons began promptly with the accelerated U.S. FDA clearance of Gilead Sciences Inc.’s oral peroxisome proliferator-activated receptor (PPAR)-delta drug, Livdelzi (seladelpar), for primary biliary cholangitis (PBC). The space includes Ipsen Pharma SA’s dual PPAR alpha/delta agonist, Iqirvo (elafibranor), licensed from Genfit SA and cleared in June 2024, as well as Ocaliva (obeticholic acid), the first-in-class farnesoid X receptor agonist from Intercept Pharmaceuticals Inc., greenlighted for PBC in May 2016.
Optimism on Wall Street proved wise and shares of Mirum Pharmaceuticals Inc. (NASDAQ:MIRM) rose accordingly to close June 17 at $33, up $7.08, or 27.3%, as the firm disclosed interim results from two phase IIb studies evaluating volixibat, an oral ileal bile acid transporter (IBAT) inhibitor, in patients with primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC). The drug proved its mettle in both liver biliary tract-stenosing indications, with no new safety signals turning up, Foster City, Calif.-based Mirum said.
Two sNDAs, one from Bristol Myers Squibb Co. (BMS) and the other from Mirum Pharmaceuticals Inc., have received U.S. FDA approval to further expand their treatment indications.
Dealmaking proved alive and well, with Sangamo Biosciences Inc. disclosing a tie-up worth as much as $1.19 billion with a subsidiary of Eli Lilly and Co., while Mirum Pharmaceuticals Inc. pledged to acquire for $445 million the bile-acid product portfolio owned by Travere Therapeutics Inc.
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.
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.