Sarepta and the FDA are meeting on Thursday to talk Duchenne muscular dystrophy once again. But this time, the atmosphere should be less tense. The joint meeting of the Pediatrics Advisory Committee and the Pediatric Ethics Subcommittee will focus on whether Sarepta's clinical trials of SRP-4045 and SRP-4053 -- two more exon-skipping technologies (at exons 45 and 53, respectively) aimed at restoring production of dystrophin -- should allow for the placement of a central venous catheter to deliver the study drug, in place of weekly infusions. The latter are too troublesome for some of the kids to handle; the controversy arose at UCLA's trial site, where three of the five kids enrolled are autistic. The ethical debate centers around whether it's okay for the placebo group to have these catheters placed, because it's not clear that the benefits would outweigh the risks in this population. The FDA's decision last year to green-light Sarepta's exon-skipping drug eteplirsen -- branded Exondys 51 for skipping exon 51 -- proved controversial.
FDA officials writing in the New England Journal of Medicine praised Neurocrine's playbook for bringing valbenazine (Ingrezza) for tardive dyskinesia to market. The take-home? "An important lesson for drug developers is that creativity and partnership in both development and review can effectively overcome long-standing clinical challenges."
First, carfentanil was the more-potent version of the opioid fentanyl that was claiming lives; now there's acryl-fentanyl. This designer drug is so new it isn't yet labeled a controlled substance by the DEA, so it's being sold -- legally -- online. (NBC News)
The U.S. may have saved billions because the FDA didn't rush to approve Eli Lilly's failed Alzheimer's drug solanezumab. (Reuters)
Ousted surgeon general Vivek Murthy blasted HHS Secretary Tom Price for comments he made about addiction treatment. Price, a former orthopedic surgeon, raised doubts about medication assisted treatment: "If we're just substituting one opioid for another, we're not moving the dial much." Murthy said it's "important that people know the truth about what science says about opioid addiction treatment: medication-assisted treatment works." (STAT News)
Neurologists are losing a wealth of information about neurological compounds, because results of trials for drugs that don't make it to market are "heavily underreported." (Annals of Neurology)