Adcomms
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.
The FDA has four big events in the coming two weeks, including an advisory panel meeting for an ultra-rare disease.
The FDA’s Oncologic Drugs Advisory Committee on Thursday strongly supported changing clinical trials for non-small cell lung cancer drugs given in the perioperative setting, pointing out the need for a more nuanced approached to treatments before and after surgery.