The FDA has approved Cobenfy, a dual M1/M4 muscarinic agonist that offers a fundamentally different approach to treating schizophrenia. The fixed dose combination of xanomeline-trospium is the first to act via a novel mechanism for the serious psychiatric disorder in over 50 years, finally expanding the treatment options beyond dopamine-targeted therapies. Bristol Myers Squibb Co., which acquired Cobenfy developer Karuna Therapeutics Inc. for $14 billion in a deal that closed in March 2024, said the drug will be available in the U.S. from late October.

Nine years on from securing $3.84 million for a phase I clinical trial to test the formulation, with results showing it overcame side effects that had confounded its forerunner, the schizophrenia treatment Karxt met its PDUFA date Sept. 26 with no decision by midday. If approved, the fixed combination of xanomeline-trospium will be the first in a new drug class, and as a dual M1/M4 muscarinic agonist, the first new therapy to act via a novel mechanism for the serious psychiatric disorder in over 50 years.

China’s National Medical Products Administration has approved Sino Biopharmaceutical Ltd.’s rivastigmine transdermal patch to treat mild to moderate Alzheimer’s disease. Developed by Sino Biopharm, the patch is the first domestically produced rivastigmine transdermal patch approved for marketing. Rivastigmine is a cholinesterase inhibitor used for the treatment of Alzheimer’s disease.

With two drugs cleared by the U.S. FDA for Niemann-Pick disease type C (NPC) in less than seven days, Wall Street was pondering the differences between the compounds, given what’s known so far about each. Most recently, the FDA approved Intrabio Inc.’s Aqneursa (levacetylleucine) on its PDUFA date for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing at least 15 kg, making Aqneursa the only approved stand-alone therapy indicated for NPC. On Sept. 20, Zevra Therapeutics Inc. won FDA clearance for Miplyffa (arimoclomol) as the first treatment for NPC.

What the results might mean for the future of other developers in the cannabinoid receptor 1 (CB1) weight-loss arena came into question after Novo Nordisk A/S unveiled phase IIa findings with monlunabant, a small-molecule oral inverse agonist, formerly INV-202.

The specter of elevated liver enzymes, a known problem with the drug class, became a topic of talk with regard to Sanofi SA’s tolebrutinib, the central nervous system-penetrating Bruton's tyrosine kinase (BTK) inhibitor for multiple sclerosis (MS). Meanwhile, another player in the space, Immunic Inc., has caught the eye of Wall Street lately.

Following the recent recommendation of its advisory committee, the U.S. FDA gave the nod to Zevra Therapeutics Inc.’s arimoclomol, a day ahead of its Sept. 21 PDUFA date, as the first treatment for Niemann-Pick disease type C (NPC), a rare genetic disease linked to progressive neurological symptoms that is almost always fatal. Branded Miplyffa, it is indicated for use in combination with enzyme inhibitor miglustat in adults and children, ages 2 and older.