When placebo data from the only randomized trial of the Duchenne muscular dystrophy gene therapy were compared with Sarepta’s proffered external control data, the placebo subjects numerically outperformed the external controls, ‘calling into question the comparability of the external control group,’ an FDA statistical reviewer said.
A US Food and Drug Administration statistician used placebo arm data from a Sarepta Therapeutics, Inc. clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) to highlight the challenges of trying to use external controls to demonstrate efficacy.
When placebo data from the only randomized, double-blind trial of the Duchenne muscular dystrophy gene therapy were compared with Sarepta’s...
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Minoryx explained to Pink Sheet why it believes its resubmission for leriglitazone stands a stronger chance of approval, and highlighted the value of early dialogue with the regulators when it comes to rare diseases, where the understanding of the natural history and outcomes is often limited.
Quick responses to late-breaking changes to US FDA COVID-19 vaccine policy kept Novavax's Nuvaxovid and Moderna's mNexspike close to expected approval timelines despite revised indications and new post-marketing trial commitments.
The once and current CBER director’s justification for his COVID-19 decision stands as a warning of the uncertainty sponsors, particularly those in the vaccine space, may now face at FDA.
Pink Sheet Drug Review Profile feature listing FDA staff who conducted the reviews of Novavax and Moderna's post-pandemic COVID-19 vaccines
Drug companies are being urged to review their internal processes to ensure compliance with new requirements in the EU’s variations guidelines, which take effect in January 2026.
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
The risks, as well as the benefits, of vaccination could be long-term, Advisory Committee on Immunization Practices member Retsef Levi said. The FDA’s Tracy Beth Høeg also said long-term safety data are needed to inform the benefit-risk analysis of the hepatitis B birth dose.
