Adcomms
Following news of RSV lower respiratory tract infections in infants immunized with Moderna’s investigational RNA vaccines, FDA advisors said the trial investigators should continue the study, while keeping an eye out for further safety signals.
The pharma is seeking full approval for its anticoagulation reversal drug Andexxa, which the FDA granted accelerated approval in 2018 for patients who had been treated with apixaban or rivaroxaban.
While an FDA advisory committee saw signals of efficacy for Lexicon Pharmaceuticals’ sotagliflozin, the panel of external experts found that the company did not provide enough data to support its drug application.
As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market.
Despite substantial variability in the presented data and no well-controlled trial, the FDA advisory committee voted in favor of Stealth BioTherapeutics’ Barth syndrome therapy elamipretide, citing the urgent unmet need.
The FDA’s reviewers pointed out that Stealth’s elamipretide missed its primary efficacy endpoints in the main study used to establish its effectiveness.
With a Friday advisory committee meeting looming, the sole indication for Intercept Pharmaceuticals’ Ocaliva appears to be at risk as the regulator flags issues regarding its post-marketing results.
The Oncologic Drugs Advisory Committee in a Sept. 26 meeting will discuss whether the regulator should restrict approval of checkpoint inhibitors based on PD-L1 expression levels.
An FDA advisory committee agreed on Friday that Zevra Therapeutics had provided sufficient efficacy data supporting the approval of arimoclomol for Niemann-Pick disease type C.
The Genetic Metabolic Diseases Advisory Committee will meet on Friday to discuss Zevra’s modified scale to describe the efficacy of its drug candidate for the treatment of Niemann-Pick disease type C.