Tag: rare diseases

And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is, so far, rather modest. We plan to catch up on our reading, promenade with the official mascots and celebrate the turning of another page on the calendar with our remaining Pharmalot ancestor. We also hope to hold another listening party, where the rotation will likely include this, this, this, this and this. And what about you? This is the time of year when apples and pumpkins grow lonely, waiting for someone to take them to a nice home. So perhaps a drive in the country is in order. This may also be an opportunity to simply enjoy the great outdoors, given the lovely change of scenery that is taking place. Or you could keep busy by strolling city streets and watching life go by. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon…

Argenx disclosed that its blockbuster drug Vyvgart failed to outperform placebo in a Phase 3 trial of patients with Sjögren’s disease, a chronic autoimmune disorder, marking an unusual setback for the company, STAT says. Argenx has racked up a series of wins in autoimmune disorders en route to becoming one of the world’s largest biotechs based on market cap. William Blair analysts had projected $1.4 billion in peak sales from Vyvgart in Sjögren’s disease but described the trial as high risk, in part because Sjögren’s patients often have high placebo responses. The trial was supposed to be completed next year, but it was stopped early after a data monitoring committee determined the primary endpoint would not be met.

The Food and Drug Administration flagged a potential safety signal after four deaths were reported among patients with treatment-resistant schizophrenia who received investigational evenamide in clinical trials, MedPage Today informs us. In April, the agency halted patient enrollment in a Phase 3 trial for the Newron Pharmaceuticals medicine after the sudden death of a participant. That hold remains in place at U.S. study centers but enrollment continues in other countries, and additional clinical sites are being added. The FDA concern relates to the risk that some sodium channel blockers may trigger arrhythmias that can lead to serious cardiovascular events or seizures.