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.

Breakthrough – or even better, revolutionary breakthrough – is perhaps the most overused term in drug development. But the discovery and development of GLP-1 receptor agonists (GLP-1RAs), which was honored with the 2024 Lasker-DeBakey Clinical Medical Research Award just last week, is one of the rare innovations that is deserving of the title.

As of the end of August 2024, the BioWorld Neurological Diseases Index (BNDI) reported a decline of 8.89%, lagging behind the Nasdaq Biotechnology Index, which gained 11.7%, and the Dow Jones Industrial Average, which rose by 7.05%. While a slight recovery from BNDI’s lowest point this year, in late April when it was down 12.2%, the index had shown modest gains over recent months before slipping again.

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.

At the 2024 European College of Neuropsychopharmacology (ECNP) Congress, researchers have presented work that could lead to ways to boost brain development and prevent neurodegeneration in individuals with Down syndrome.

Chimeric antigen receptor (CAR) T-cell therapy moved the needle at the 2024 ECTRIMS (European Committee for Treatment and Research in Multiple Sclerosis) congress and will continue to do so – not just in multiple sclerosis (MS), but in other autoimmune diseases as well.