The risk and benefit of Pfizer Inc.’s oral sickle cell disease drug Oxbryta (voxelotor) has flipped, prompted by what the company called new clinical data indicating “an imbalance in vaso-occlusive crises and fatal events” that need more study. Based on an EMA recommendation, 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.
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.
Japan’s Ministry of Health, Labour and Welfare 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.
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Acroimmune, Asklepios, Aitia, Bavarian Nordic, Bayer, Belief, Emergent, Mitem, Novartis, Oncoc4, Orion, Zetagen.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: 2seventy Bio, Abbvie, Arcutis, Avadel, Biocon, Bristol Myers, Eli Lilly, Ferring, Galderma, Inflarx, Merck, UCB, Windtree.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Amarex, Amgen, Eli Lilly, Elixirgen, Heron, Junshi, Merck.
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.
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