Years after approving three glucagon-like peptide-1 (GLP-1) receptor agonists to treat obesity, the U.S. FDA is acknowledging that the drugs don’t have the same risk seen with older weight-loss medicines. Citing its post-market evaluation that found no increased risk of suicidal ideation or behavior, the U.S. FDA is requesting that the risk be removed from the warnings and precautions section of labeling for the GLP-1 obesity drugs – Eli Lilly and Co.’s Zepbound (tirzepatide) and Novo Nordisk A/S’ Saxenda (liraglutide) and Wegovy (semaglutide).
Dentatorubral-pallidoluysian atrophy (DRPLA) is a lethal neurodegenerative disorder caused by pathogenic expansion of CAG repeats within the atrophin-1 (ATN1) gene. As DRPLA belongs to the broader class of repeat expansion disorders (RED) that are driven by toxic gain-of-function effects, reduction or elimination of ATN1 expression is predicted to provide therapeutic benefit.
Perhaps the biggest indicator of U.S. President Donald Trump’s activism in his second term is the 225 executive orders (EOs) he issued in 2025. The pace of those orders seems to have slowed, with “only” 16 released in the last quarter of the year. Four of the recent EOs could impact drug and device companies in a myriad of ways.
The U.S. FDA has approved Zycubo (copper histidinate) as the first treatment for Menkes disease, a rare, genetic disease affecting children who cannot absorb copper through their intestines, leading to seizures, weak muscles, a failure to thrive and, ultimately, if left untreated, an early death by age 3.
Researchers at Iama Therapeutics Srl and Italian Institute of Technology have disclosed solute carrier family 12 member 2 (SLC12A2; NKCC1) inhibitors reported to be useful for the treatment of Down syndrome.
Neurotrimin (NTM) is a member of the IgLON family, the disruption of which has been tied to emotional learning deficits and anxiety-like behavior in animal models. A mutation in the NTM gene was found to disrupt NTM protein heterodimerization with other IgLON family members, suggesting a potential link between NTM dysfunction and neurodevelopmental and behavioral disorders.
With rumors regarding a couple of potential mega-mergers making the rounds, the week of the annual J.P. Morgan Healthcare Conference kicked off with the official disclosure of some billion-dollar collaborations, leading with Abbvie Inc.’s exclusive licensing deal with Remegen Co. Ltd. for PD-1/VEGF-targeted bispecific antibody RC-148.
Stoke Therapeutics Inc.’s speeded-up timeline for zorevunersen, the antisense oligonucleotide in development with Biogen Inc. as a first-in-class potential disease-modifying treatment for Dravet syndrome, put the rare, severe form of lifelong epilepsy in the spotlight. The news involved completion of enrollment and a phase III data readout from the Emperor study, as officials said signups of 150 patients are expected in the second quarter of the year, which puts the study on track for data in mid-2027.
For individuals with major depressive disorder (MDD) that does not respond to pharmacotherapy, recent decisions by the U.S. FDA enabling at-home use of neuromodulation devices may provide a bright spot in dark days. Neurolief Ltd. received FDA premarket approval for Proliv Rx, a physician-directed, brain neuromodulation therapy indicated for use as an at-home adjunctive treatment for adults with MDD who failed to respond adequately to at least one previous antidepressant on Jan. 7. That decision follows the December 510(k) clearance for at-home use of the Flow brain-stimulation device by Flow Neuroscience AB as either a monotherapy or as an adjunctive treatment.
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