Good morning, everyone, and how are you today? We are doing just fine, thank you, despite the cloudy skies looming over the Pharmalot campus. After all, the birds are still chirping and a cool breeze is wafting by. Moreover, this marks the middle of the week, which means we have managed to survive this far. And this calls for celebration, yes? So please join us as we hoist another cup of delicious stimulation. Our choice today is maple cinnamon French toast, a yummy addition to the pantry. And of course, no prescription is required. Meanwhile, here are a few items of interest. Have a grand day and drop us a line if you hear something saucy. …
Sarepta Therapeutics will restart a clinical trial previously put on hold over safety concerns after reaching an agreement with the U.S. Food and Drug Administration on changes to the study’s plan, BioPharma Dive writes. Moving forward, the company will expand patient monitoring in the trial to watch for low magnesium levels, which had resulted in a serious adverse reaction in one study participant that led the FDA to suspend testing earlier this year. The drug being tested, called SRP-5051, treats Duchenne muscular dystrophy and is meant to be a more potent successor to Sarepta’s approved medicine Exondys 51.
Even with new data, FDA reviewers remain skeptical about an investigational drug for ALS that is getting a rare second chance before an agency advisory committee on Wednesday, MedPage Today says. Post hoc analyses submitted by Amylyx Pharmaceuticals do not appear to provide “independent confirmatory evidence” of the clinical effect of the drug, called AMX0035 in ALS, FDA reviewers wrote in a briefing document. The Wednesday meeting marks the second time the drug will go before the agency’s Peripheral and Central Nervous System Drugs Advisory Committee. Read more in The Washington Post.
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