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. “We really started with a blank sheet of paper,” said the main inventor of Karxt, Eric Elenko, president of Puretech Health plc, which founded developer Karuna Therapeutics Inc. “We were interested in schizophrenia because it’s a devastating disease and if you look at existing drugs, they can often leave residual symptoms, have bad adverse events, and there really hasn’t been a truly innovative drug in decades,” he told BioWorld.

Pfizer pulls sickle cell drug after deaths and health complications

The risk and benefit of Pfizer Inc.’s oral sickle cell disease drug Oxbryta (voxelotor) has flipped, prompted by what the company called clinical data indicating “an imbalance in vaso-occlusive crises and fatal events” that need more study. Pfizer said it is voluntarily recalling all lots of Oxbryta from wherever it’s approved worldwide. Pfizer also is shuttering its Oxbryta clinical studies and expanded access programs. The EMA’s human medicines committee now has recommended suspending the treatment’s marketing authorization. Oxbryta, which inhibits sickle hemoglobin polymerization to treat the hemolytic anemia caused by sickle cell disease, came to Pfizer in 2022 through the $5.4 billion acquisition of Global Blood Therapeutics Inc.

FDA's ODAC eyes PD-L1 expression in immune checkpoint inhibitors

The FDA’s Oncologic Drugs Advisory Committee (ODAC) is meeting in a day-long session to decide whether approval of immune checkpoint inhibitors should be restricted with an eye to PD-L1 expression levels. Involved are Bristol Myers Squibb Co.’s Opdivo (nivolumab, given the first go-ahead in December 2014) and Yervoy (ipilimumab, first cleared in March 2011), along with Merck & Co.’s Keytruda (pembrolizumab, greenlighted in September 2014). The panel is scrutinizing Beigene Ltd.’s BLA related to Tevimbra (tislelizumab), too. ODAC has been asked to weigh in on a list of matters that includes the adequacy of PD-L1 expression as a predictive biomarker, along with the differing risk-benefit assessments in different subpopulations defined by PD-L1 expression, and the adequacy of the cumulative data to restrict the approvals of immune checkpoint inhibitors based on PD-L1.

New Japan drug approvals: cancer, insomnia, Alzheimer’s and more

Japan’s Ministry of Health, Labour and Welfare (MHLW) granted new drug approvals and expanded indications for conditions like cancer, insomnia and Alzheimer’s disease (AD) Sept. 24, including Eli Lilly and Co.’s Kisunla (donanemab) for early symptomatic AD. The MHLW also granted an additional indication to Tokyo-based Otsuka Pharmaceutical Co. Ltd.’s Rexulti (brexpiprazole) to treat symptoms of agitation associated with AD. And it gave regulatory nods to Nxera Pharma Japan Co. Ltd., formerly Sosei Group, for Quviviq (daridorexant) to treat insomnia, and to Fruzaqla (fruquintinib), Padcev (enfortumab vedotin) and Keytruda (pembrolizumab) for cancer indications.

China approves Sino Biopharm’s patch for Alzheimer’s disease

China’s National Medical Products Administration (NMPA) 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. Acetylcholine is a neurotransmitter that transmits messages between neurons and plays a key role in brain regions associated with memory and learning. Rivastigmine reduces the breakdown of acetylcholine by inhibiting the activity of acetylcholinesterase and butyrylcholinesterase.

ECNP 2024: GLP-1 drugs could have mental health benefits

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. In fact, the merits of drugs such as Mounjaro and Ozempic are probably one of the few topics that could unite attendees of 2024 Fall Fashion Week and the 2024 European College of Neuropsychopharmacology (ECNP) Congress, both of which are being held in Milan this week. Riccardo De Giorgi, clinical lecturer in general adult psychiatry at Oxford University, poked fun at the connection in his presentation, when he said that GLP-1RAs had come to the attention of “very important people. Like one of the Kardashian sisters – don’t ask me which one.”

US Congress avoids government shutdown, extends voucher program

Both chambers of the U.S. Congress put aside their election year politicking long enough yesterday to pass a continuing resolution (CR) that will keep the government running at its current funding level through Dec. 20. Now awaiting President Joe Biden’s signature, the CR also extends the FDA’s pediatric rare disease priority review voucher (PRV) program for three more months. Without the extension, the PRV program would have died with fiscal 2024 at midnight Sept. 24.

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